Zobrazeno 1 - 10
of 448
pro vyhledávání: '"Sancricca, C"'
Autor:
Gorgoglione D; Department of Neurosciences, Neuromuscular Center, University of Padova, 35128 Padova, Italy., Sabbatini D; Department of Neurosciences, Neuromuscular Center, University of Padova, 35128 Padova, Italy.; Unit of Biostatistics, Epidemiology and Public Health, Department of Cardiac, Thoracic, Vascular Sciences, and Public Health, University of Padova, 35131 Padova, Italy., Riguzzi P; Department of Neurosciences, Neuromuscular Center, University of Padova, 35128 Padova, Italy., Capece G; Department of Neurosciences, Neuromuscular Center, University of Padova, 35128 Padova, Italy., Pane M; Pediatric Neurology, Università Cattolica del Sacro Cuore, 00168 Roma, Italy.; Centro Clinico Nemo Pediatrico, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Roma, Italy., Servidei S; Neurophysiopathology unit Fondazione Policlinico Universitario A. Gemelli IRCCS, 00168 Rome, Italy.; Dipartimento Universitario di Neuroscienze, Università Cattolica del Sacro Cuore, 00168 Rome, Italy., Briganti M; Neurophysiopathology unit Fondazione Policlinico Universitario A. Gemelli IRCCS, 00168 Rome, Italy.; Fondazione UILDM Lazio Onlus, 00167 Rome, Italy., Sancricca C; Neurophysiopathology unit Fondazione Policlinico Universitario A. Gemelli IRCCS, 00168 Rome, Italy.; Fondazione UILDM Lazio Onlus, 00167 Rome, Italy., Bruschi F; Department of Pediatric Neuroscience, Fondazione IRCCS Istituto Neurologico Carlo Besta, 20133 Milan, Italy., Ardissone A; Department of Pediatric Neuroscience, Fondazione IRCCS Istituto Neurologico Carlo Besta, 20133 Milan, Italy., Masson R; Department of Pediatric Neuroscience, Fondazione IRCCS Istituto Neurologico Carlo Besta, 20133 Milan, Italy., Gallone A; Neuroimmunology and Neuromuscular Diseases Unit, Fondazione IRCCS Istituto Neurologico 'Carlo Besta', 20133 Milan, Italy., Maggi L; Neuroimmunology and Neuromuscular Diseases Unit, Fondazione IRCCS Istituto Neurologico 'Carlo Besta', 20133 Milan, Italy., Picillo E; Medical Genetics and Cardiomyology, University of Campania 'Luigi Vanvitelli', 80138 Napoli, Italy., Politano L; Cardiomyology and Medical Genetics, University of Campania 'Luigi Vanvitelli', 80138 Napoli, Italy., Petrosino A; Department of Neurosciences, Neuromuscular Center, University of Padova, 35128 Padova, Italy., Vianello S; Department of Neurosciences, Neuromuscular Center, University of Padova, 35128 Padova, Italy., Penzo M; Department of Neurosciences, Neuromuscular Center, University of Padova, 35128 Padova, Italy., Villa M; Department of Neurosciences, Neuromuscular Center, University of Padova, 35128 Padova, Italy., Sframeli M; Department of Clinical and Experimental Medicine, University of Messina, 98122 Messina, Italy., Allegra C; Department of Clinical and Experimental Medicine, University of Messina, 98122 Messina, Italy., Barp A; The NEMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, 20162 Milan, Italy., Di Bari A; The NEMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, 20162 Milan, Italy., Salmin F; The NEMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, 20162 Milan, Italy., Albamonte E; The NEMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, 20162 Milan, Italy., Colacicco G; The NEMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, 20162 Milan, Italy., Panicucci C; Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, 16147 Genova, Italy., Traverso M; Pediatric Neurology and Muscle Disease Unit, IRCCS Istituto Giannina Gaslini, 16147 Genova, Italy., Palermo C; Centro Clinico Nemo Pediatrico, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Roma, Italy., Lerario A; Neuromuscular and Rare Disease Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, 20122 Milano, Italy., Velardo D; Neuromuscular and Rare Disease Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, 20122 Milano, Italy., D'Angelo MG; Unit of Rare Diseases of the Central and Peripheral Nervous System, Scientific Institute IRCCS Eugenio Medea, 23842 Bosisio Parini, Italy., Berardinelli A; IRCCS Mondino Foundation, 27100 Pavia, Italy., Gardani A; IRCCS Mondino Foundation, 27100 Pavia, Italy., Nicotra R; Child and Adolescence Neurological Unit, National Neurological Institute Casimiro Mondino Foundation, IRCCS, IRCCS Mondino Foundation, 27100 Pavia, Italy.; Department of Brain and Behavioral Sciences, University of Pavia, 27100 Pavia, Italy., Parravicini S; Child and Adolescence Neurological Unit, National Neurological Institute Casimiro Mondino Foundation, IRCCS, IRCCS Mondino Foundation, 27100 Pavia, Italy.; Department of Brain and Behavioral Sciences, University of Pavia, 27100 Pavia, Italy., Siciliano G; Department of Clinical and Experimental Medicine, University of Pisa, 56126 Pisa, Italy., Ricci G; Department of Clinical and Experimental Medicine, University of Pisa, 56126 Pisa, Italy., Torri F; Department of Clinical and Experimental Medicine, University of Pisa, 56126 Pisa, Italy., Gadaleta G; Neuromuscular Unit, Department of Neurosciences RLM, University of Torino, 10126 Turin, Italy., Urbano G; Neuromuscular Unit, Department of Neurosciences RLM, University of Torino, 10126 Turin, Italy., Rolle E; Neuromuscular Unit, Department of Neurosciences RLM, University of Torino, 10126 Turin, Italy., Ricci F; Neuromuscular Unit, Department of Neurosciences RLM, University of Torino, 10126 Turin, Italy., D'Amico A; IRCCS, Unit of Muscular and Neurodegenerative disorders, Bambino Gesù Children's Hospital, 00165 Rome, Italy., Catteruccia M; IRCCS, Unit of Muscular and Neurodegenerative disorders, Bambino Gesù Children's Hospital, 00165 Rome, Italy., Pini A; IRCCS Istituto delle Scienze Neurologiche di Bologna, Neuromuscular Pediatric Unit, 40139 Bologna, Italy., Giannotta M; IRCCS Istituto delle Scienze Neurologiche di Bologna, Neuromuscular Pediatric Unit, 40139 Bologna, Italy., Battini R; Department of Clinical and Experimental Medicine, University of Pisa, 56126 Pisa, Italy.; Department of Developmental Neuroscience, IRCCS Fondazione Stella Maris, 56128 Pisa, Italy., Marinella G; Department of Developmental Neuroscience, IRCCS Fondazione Stella Maris, 56128 Pisa, Italy., Previtali SC; Neuromuscular Repair Unit, Institute of Experimental Neurology (InSpe), Division of Neuroscience, IRCCS Ospedale San Raffaele, 20132 Milan, Italy., Zambon AA; Neuromuscular Repair Unit, Institute of Experimental Neurology (InSpe), Division of Neuroscience, IRCCS Ospedale San Raffaele, 20132 Milan, Italy., Ferlini A; Unit of Medical Genetics, Department of Medical Sciences, University of Ferrara, 44121 Ferrara, Italy., Fortunato F; Unit of Medical Genetics, Department of Medical Sciences, University of Ferrara, 44121 Ferrara, Italy., Magri F; Neurology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, 20122 Milan, Italy., Mongini TE; Neuromuscular Unit, Department of Neurosciences RLM, University of Torino, 10126 Turin, Italy., Sansone VA; The NEMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, 20162 Milan, Italy., Bruno C; Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, 16147 Genova, Italy.; Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health-DINOGMI, University of Genova, 16132 Genova, Italy., Messina S; Department of Clinical and Experimental Medicine, University of Messina, 98122 Messina, Italy., Nigro V; Medical Genetics and Cardiomyology, University of Campania 'Luigi Vanvitelli', 80138 Napoli, Italy.; Telethon Institute of Genetics and Medicine, 80078, Italy., Moroni I; Department of Pediatric Neuroscience, Fondazione IRCCS Istituto Neurologico Carlo Besta, 20133 Milan, Italy., Mercuri E; Pediatric Neurology, Università Cattolica del Sacro Cuore, 00168 Roma, Italy.; Centro Clinico Nemo Pediatrico, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Roma, Italy., Bello L; Department of Neurosciences, Neuromuscular Center, University of Padova, 35128 Padova, Italy., Pegoraro E; Department of Neurosciences, Neuromuscular Center, University of Padova, 35128 Padova, Italy.
Publikováno v:
Brain : a journal of neurology [Brain] 2024 Nov 05. Date of Electronic Publication: 2024 Nov 05.
Autor:
Strafella C; Genomic Medicine Laboratory UILDM, IRCCS Santa Lucia Foundation, Via Ardeatina 306-354, 00179, Rome, Italy., Megalizzi D; Genomic Medicine Laboratory UILDM, IRCCS Santa Lucia Foundation, Via Ardeatina 306-354, 00179, Rome, Italy.; Department of Biomedicine and Prevention, Tor Vergata University of Rome, Via Montpellier 1, 00133, Rome, Italy., Trastulli G; Genomic Medicine Laboratory UILDM, IRCCS Santa Lucia Foundation, Via Ardeatina 306-354, 00179, Rome, Italy.; Department of System Medicine, Tor Vergata University of Rome, Via Montpellier 1, 00133, Rome, Italy., Proietti Piorgo E; Genomic Medicine Laboratory UILDM, IRCCS Santa Lucia Foundation, Via Ardeatina 306-354, 00179, Rome, Italy., Colantoni L; Genomic Medicine Laboratory UILDM, IRCCS Santa Lucia Foundation, Via Ardeatina 306-354, 00179, Rome, Italy., Tasca G; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, NE1 3BZ, UK., Monforte M; Unità Operativa Complessa di Neurologia, Fondazione Policlinico Universitario A. Gemelli IRCCS, Largo Agostino Gemelli 8, 00168, Rome, Italy., Zampatti S; Genomic Medicine Laboratory UILDM, IRCCS Santa Lucia Foundation, Via Ardeatina 306-354, 00179, Rome, Italy., Primiano G; Neurofisiopathology Unit, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Largo Agostino Gemelli 8, 00168, Rome, Italy., Sancricca C; Neurofisiopathology Unit, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Largo Agostino Gemelli 8, 00168, Rome, Italy., Bortolani S; Unità Operativa Complessa di Neurologia, Fondazione Policlinico Universitario A. Gemelli IRCCS, Largo Agostino Gemelli 8, 00168, Rome, Italy., Torchia E; Unità Operativa Complessa di Neurologia, Fondazione Policlinico Universitario A. Gemelli IRCCS, Largo Agostino Gemelli 8, 00168, Rome, Italy., Ravera B; Unità Operativa Complessa di Neurologia, Fondazione Policlinico Universitario A. Gemelli IRCCS, Largo Agostino Gemelli 8, 00168, Rome, Italy., Torri F; Department of Clinical and Experimental Medicine, University of Pisa, Via Roma 55, 56126, Pisa, Italy., Gadaleta G; Presidio Molinette e OIRM (SS Malattie Neuromuscolari e SC Neuropsichiatria Infantile), AOU Città della Salute e della Scienza di Torino, Corso Bramante 88, 10126, Turin, Italy., Risi B; NeMO-Brescia Clinical Center for Neuromuscular Diseases, Via Paolo Richiedei, 16, 25064, Brescia, Italy., Caria F; NeMO-Brescia Clinical Center for Neuromuscular Diseases, Via Paolo Richiedei, 16, 25064, Brescia, Italy., Gerardi F; The NEMO Center in Milan, Neurorehabilitation Unit,, University of Milan, ASST Niguarda Hospital, Piazza Dell'Ospedale Maggiore 3, 20161, Milan, Italy., Carraro E; The NEMO Center in Milan, Neurorehabilitation Unit,, University of Milan, ASST Niguarda Hospital, Piazza Dell'Ospedale Maggiore 3, 20161, Milan, Italy., Gioiosa V; Department of Medico-Surgical Sciences and Biotechnologies, University of Rome Sapienza, Corso Della Repubblica 79, 4100, Latina, Italy., Garibaldi M; Neuromuscular and Rare Disease Centre, Sant'Andrea Hospital, Via Di Grottarossa 1035-1039, 00189, Rome, Italy.; Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), Faculty of Medicine and Psychology, SAPIENZA University of Rome, Piazzale Aldo Moro 5, 00185, Rome, Italy., Tufano L; Neuromuscular and Rare Disease Centre, Sant'Andrea Hospital, Via Di Grottarossa 1035-1039, 00189, Rome, Italy.; Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), Faculty of Medicine and Psychology, SAPIENZA University of Rome, Piazzale Aldo Moro 5, 00185, Rome, Italy., Frezza E; Department of Systems Medicine, Neurology Unit, Tor Vergata University of Rome, Via Montpellier 1, 00133, Rome, Italy., Massa R; Department of Systems Medicine, Neurology Unit, Tor Vergata University of Rome, Via Montpellier 1, 00133, Rome, Italy., Caltagirone C; Department of Clinical and Behavioral Neurology, IRCCS Fondazione Santa Lucia, Via Ardeatina 306-354, 00179, Rome, Italy., Pennisi EM; UOC of Neurology, San Filippo Neri Hospital, Via Giovanni Martinotti 20, 00135, Rome, Italy., Petrucci A; Department of Neurology and Neurophysiopathology, Azienda Ospedaliera San Camillo Forlanini, Circonvallazione Gianicolense, 87, 00149, Rome, Italy., Pane M; Pediatric Neurology, Università Cattolica del Sacro Cuore, Largo Francesco Vito 1, 00168, Rome, Italy.; Centro Clinico Nemo, Fondazione Agostino Gemelli IRCCS, Largo Agostino Gemelli 8, 00168, Rome, Italy., Frongia A; Centro Clinico Nemo, Fondazione Agostino Gemelli IRCCS, Largo Agostino Gemelli 8, 00168, Rome, Italy., Gragnani F; Neurology and Neurophysiopathology Unit, Sandro Pertini Hospital, Via Dei Monti Tiburtini 385, 00157, Rome, Italy., Scutifero M; Cardiomyology and Medical Genetics, University of Campania Luigi Vanvitelli, Via Santa Maria Di Costantinopoli 16, 80138, Naples, Italy., Mandich P; IRCCS Ospedale Policlinico San Martino - UOC Genetica Medica, Largo R. Benzi 10, 16132, Genoa, Italy.; Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics and Maternal and Child Health, University of Genova, Largo Paolo Daneo 3, 16132, Genoa, Italy., Grandis M; IRCCS Ospedale Policlinico San Martino - UOC Genetica Medica, Largo R. Benzi 10, 16132, Genoa, Italy.; Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics and Maternal and Child Health, University of Genova, Largo Paolo Daneo 3, 16132, Genoa, Italy., Maioli MA; Centro Sclerosi Multipla, ASSL, Via Is Guadazzonis 2, P.O. Binaghi, 09126, Cagliari, Italy., Casali C; Department of Medico-Surgical Sciences and Biotechnologies, University of Rome Sapienza, Corso Della Repubblica 79, 4100, Latina, Italy., Manfroi E; Department of Neuroscience- Neurogenetics, Santa Maria Hospital, Viale Tristano Di Joannuccio, 05100, Terni, Italy., Politano L; Cardiomyology and Medical Genetics, University of Campania Luigi Vanvitelli, Via Santa Maria Di Costantinopoli 16, 80138, Naples, Italy., Passamano L; Cardiomyology and Medical Genetics, University of Campania Luigi Vanvitelli, Via Santa Maria Di Costantinopoli 16, 80138, Naples, Italy., Petillo R; Medical and Laboratory Genetics Unit, A.O.R.N. 'Antonio Cardarelli', Via A. Cardarelli 9, 80131, Naples, Italy., Rodolico C; Department of Clinical and Experimental Medicine, University of Messina, Piazza Pugliatti 1, 98122, Messina, Italy., Pugliese A; Department of Clinical and Experimental Medicine, University of Messina, Piazza Pugliatti 1, 98122, Messina, Italy., Previtali SC; Institute of Experimental Neurology (INSPE), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Via Olgettina 60, 20132, Milan, Italy., Sansone V; The NEMO Center in Milan, Neurorehabilitation Unit,, University of Milan, ASST Niguarda Hospital, Piazza Dell'Ospedale Maggiore 3, 20161, Milan, Italy., Vercelli L; Presidio Molinette e OIRM (SS Malattie Neuromuscolari e SC Neuropsichiatria Infantile), AOU Città della Salute e della Scienza di Torino, Corso Bramante 88, 10126, Turin, Italy., Mongini TE; Presidio Molinette e OIRM (SS Malattie Neuromuscolari e SC Neuropsichiatria Infantile), AOU Città della Salute e della Scienza di Torino, Corso Bramante 88, 10126, Turin, Italy., Ricci G; Department of Clinical and Experimental Medicine, University of Pisa, Via Roma 55, 56126, Pisa, Italy., Siciliano G; Department of Clinical and Experimental Medicine, University of Pisa, Via Roma 55, 56126, Pisa, Italy., Filosto M; NeMO-Brescia Clinical Center for Neuromuscular Diseases, Via Paolo Richiedei, 16, 25064, Brescia, Italy.; Department of Clinical and Experimental Sciences, University of Brescia, Viale Europa 11, 25123, Brescia, Italy., Ricci E; Unità Operativa Complessa di Neurologia, Fondazione Policlinico Universitario A. Gemelli IRCCS, Largo Agostino Gemelli 8, 00168, Rome, Italy.; Istituto Di Neurologia, Università Cattolica del Sacro Cuore, Largo Francesco Vito 1, 00168, Rome, Italy., Cascella R; Genomic Medicine Laboratory UILDM, IRCCS Santa Lucia Foundation, Via Ardeatina 306-354, 00179, Rome, Italy.; Department of Biomedical Sciences, Catholic University Our Lady of Good Counsel, Sheshi Nënë Tereza 4, 1010, Tiranë, Albania., Giardina E; Genomic Medicine Laboratory UILDM, IRCCS Santa Lucia Foundation, Via Ardeatina 306-354, 00179, Rome, Italy. emiliano.giardina@uniroma2.it.; Department of Biomedicine and Prevention, Tor Vergata University of Rome, Via Montpellier 1, 00133, Rome, Italy. emiliano.giardina@uniroma2.it.
Publikováno v:
Clinical epigenetics [Clin Epigenetics] 2024 Oct 22; Vol. 16 (1), pp. 148. Date of Electronic Publication: 2024 Oct 22.
Autor:
Megalizzi D; Genomic Medicine Laboratory UILDM, IRCCS Fondazione Santa Lucia, Via Ardeatina 306-354, 00179 Rome, Italy.; Department of Biomedicine and Prevention, Tor Vergata University of Rome, Via Montpellier 1, 00133 Rome, Italy., Trastulli G; Genomic Medicine Laboratory UILDM, IRCCS Fondazione Santa Lucia, Via Ardeatina 306-354, 00179 Rome, Italy.; Department of System Medicine, Tor Vergata University of Rome, Via Montpellier 1, 00133 Rome, Italy., Colantoni L; Genomic Medicine Laboratory UILDM, IRCCS Fondazione Santa Lucia, Via Ardeatina 306-354, 00179 Rome, Italy., Proietti Piorgo E; Genomic Medicine Laboratory UILDM, IRCCS Fondazione Santa Lucia, Via Ardeatina 306-354, 00179 Rome, Italy., Primiano G; Neurophysiopathology Unit, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Largo Agostino Gemelli 8, 00168 Rome, Italy., Sancricca C; Neurophysiopathology Unit, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Largo Agostino Gemelli 8, 00168 Rome, Italy., Caltagirone C; Department of Clinical and Behavioral Neurology, IRCCS Fondazione Santa Lucia, Via Ardeatina 306-354, 00179 Rome, Italy., Cascella R; Genomic Medicine Laboratory UILDM, IRCCS Fondazione Santa Lucia, Via Ardeatina 306-354, 00179 Rome, Italy.; Department of Chemical-Toxicological and Pharmacological Evaluation of Drugs, Catholic University Our Lady of Good Counsel, 1000 Tirana, Albania., Strafella C; Genomic Medicine Laboratory UILDM, IRCCS Fondazione Santa Lucia, Via Ardeatina 306-354, 00179 Rome, Italy., Giardina E; Genomic Medicine Laboratory UILDM, IRCCS Fondazione Santa Lucia, Via Ardeatina 306-354, 00179 Rome, Italy.; Department of Biomedicine and Prevention, Tor Vergata University of Rome, Via Montpellier 1, 00133 Rome, Italy.
Publikováno v:
International journal of molecular sciences [Int J Mol Sci] 2024 Oct 11; Vol. 25 (20). Date of Electronic Publication: 2024 Oct 11.
Autor:
Bortolani S; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Savarese M; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Vattemi G; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Bonanno S; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Falzone YM; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Pugliese A; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Primiano G; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Sancricca C; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Lopergolo D; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Greco G; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Gemelli C; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Ravaglia S; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Bencivenga RP; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Velardo D; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Magri F; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Valentino ML; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Cheli M; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Torchia E; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Lucchini M; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Petrucci A; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Ricci G; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Garibaldi M; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Astrea G; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Rubegni A; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Angelini CI; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Ariatti A; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Santorelli FM; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Ruggieri A; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Antonini G; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Siciliano G; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Filosto M; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Mirabella M; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Liguori R; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Comi GP; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Ruggiero L; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Grandis M; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Massa R; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Malandrini A; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Servidei S; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Mongini TE; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Rodolico C; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Toscano A; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Previtali SC; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Tonin P; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Diaz-Manera J; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Monforte M; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Ricci E; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Maggi L; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom., Tasca G; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.
Publikováno v:
Neurology [Neurology] 2024 Aug 27; Vol. 103 (4), pp. e209697. Date of Electronic Publication: 2024 Aug 05.
Autor:
Strafella C; Genomic Medicine Laboratory UILDM, IRCCS Santa Lucia Foundation, Rome, Italy., Colantoni L; Genomic Medicine Laboratory UILDM, IRCCS Santa Lucia Foundation, Rome, Italy., Megalizzi D; Genomic Medicine Laboratory UILDM, IRCCS Santa Lucia Foundation, Rome, Italy., Trastulli G; Genomic Medicine Laboratory UILDM, IRCCS Santa Lucia Foundation, Rome, Italy., Piorgo EP; Genomic Medicine Laboratory UILDM, IRCCS Santa Lucia Foundation, Rome, Italy., Primiano G; Neurofisiopathology Unit, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Sancricca C; Neurofisiopathology Unit, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Ricci G; Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy., Siciliano G; Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy., Caltagirone C; Department of Clinical and Behavioral Neurology, IRCCS Fondazione Santa Lucia, Rome, Italy., Filosto M; Department of Clinical and Experimental Sciences, University of Brescia, NeMO-Brescia Clinical Center for Neuromuscular Diseases, Brescia, Italy., Tasca G; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, UK., Ricci E; Unità Operativa Complessa di Neurologia, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy.; Istituto di Neurologia, Università Cattolica del Sacro Cuore, Rome, Italy., Cascella R; Genomic Medicine Laboratory UILDM, IRCCS Santa Lucia Foundation, Rome, Italy.; Department of Biomedical Sciences, Catholic University Our Lady of Good Counsel, Tirana, Albania., Giardina E; Genomic Medicine Laboratory UILDM, IRCCS Santa Lucia Foundation, Rome, Italy.; Department of Biomedicine and Prevention, Medical Genetics Laboratory, Tor Vergata University, Rome, Italy.
Publikováno v:
Clinical genetics [Clin Genet] 2024 Mar; Vol. 105 (3), pp. 335-339. Date of Electronic Publication: 2023 Dec 02.
Autor:
Albamonte, E., Coratti, G., Salmin, F., Zanolini, A., Pane, M., Pera, MC., Leone, D., Antonaci, L., D’Amico, A., Catteruccia, M., Bertini, E., Bruno, C., Brolatti, N., Messina, S., Sframeli, M., Piras, R., Mercuri, E., Sansone, V.A., Bonanno, S., Giossi, R., Zanin, R., Porcelli, V., Ingenito, G., Stevic, Z., Peric, S., Maggi, L., Darras, B.T., Masson, R., Mazurkiewicz-Bełdzińska, M., Rose, K., Xiong, H., Zanoteli, E., Baranello, G., Vlodavets, D., Dodman, A., El-Khairi, M., Gerber, M., Gorni, K., Kletzl, H., Scalco, R.S., Servais, L., Bello, L., Govoni, A., Caponnetto, C., Grisanti, S.G., Passamano, L., Grandis, M., Nicocia, G., Trojsi, F., Cerri, F., Gardani, A., Risi, B., Gadaleta, G., Ferraro, M., Bozzoni, V., Caumo, L., Tanel, R., Saccani, E., Meneri, M., Vacchiano, V., Ricci, G., Sorarù, G., D’Errico, E., Maioli, M.A., Tramacere, I., Bortolani, S., Pavesi, G., Silvestrini, M., Politano, L., Schenone, A., Previtali, S.C., Berardinelli, A., Turri, M., Verriello, L., Coccia, M., Mantegazza, R., Liguori, R., Filosto, M., Marrosu, G., Siciliano, G., Simone, I.L., Mongini, T., Comi, G.P., Pegoraro, E., Mercante, A., Perilongo, G., Salamon, E., Santini, A., Benini, F., Agosto, C., Cotti Piccinelli, S., Pilotto, A., Cristillo, V., Schiano di Cola, F., Bonzi, G., Mazzola, M., Padovani, A., Nuredini, A., Taiana, M., Nizzardo, M., Mari, F., Cesaroni, E., Porfiri, L., Tiziano, D., Vita, G.L., Merico, E., Schirinzi, E., Ramos, J.A.F., Ostrowska, I., Piontek, M., Corti, S., Miller, W., Shieh, P.B., Kuntz, N., Dowling, J.J., Müller-Felber, W., Blaschek, A., Bönnemann, C.G., Foley, A.R., Saade, D.N., Seferian, A.M., Lawlor, M.W., Noursalehi, M., Prasad, S., Rico, S., Rodino-Klapac, L.R., Pozsgai, E.R., Lewis, S., Griffin, D.A., Meadows, A.S., Lehman, K.J., Church, K., Reash, N.F., Iammarino, M.A., Powers, B., Alfano, L.N., Lowes, L.P., Koenig, E., Neuhaus, S., Li, X., Picaro, L., Mendell, J.R., Tonacci, A., Torri, F., Aringhieri, G., Sansone, F., Rubegni, A., Santorelli, F.M., Conte, R., Riguzzi, P., Zangaro, V., Villa, F.M., Cestarollo, M., Savarese, M., Johari, M., Vihola, A., Luque, H., Hackman, P., Udd, B., Bianchi, F., Fortunato, F., Giannini, F., Malandrini, A., Silani, V., Ticozzi, N., Fenu, S., Peduto, C., D’Ambrosio, P., Primiano, G., Sancricca, C., Sciacco, M., Brusa, R., Solero, L., Brusa, C., Cateruccia, M., Diodado, D., Pugliese, A., Ferlini, A., Monastra, F.M., Blasevich, F., Bragato, C., Marinella, G., Buchignani, B., Astrea, G., Cassandrini, D., Donati, M.A., Gallone, S., Lopergolo, D., Maioli, MA., Magri, F., Mandich, P., Massa, R., Matà, S., Moggio, M., Mongini, T.E., Ricci, F., Rodolico, C., Sperti, M., Ticci, C., Tonin, P., Battini, R., Panicucci, C., Pedemonte, M., Casalini, E., Minetti, C., Maghnie, M., Di Iorgi, N., Sabbatini, D., Vianello, S., Fusto, A., Merlo, B., Parravicini, S., Travaglioni, L., D’Angelo, G., Di Bari, A., Canioni, E., Gallone, A., Picillo, E., Nigro, V., Trucco, F., Ridout, D., Maresh, K., Chesshyre, M., Munot, P., Sarkozy, A., Robb, S., Quinlivan, R., Riley, M., Wallis, C., Chan, E., Abel, F., De Lucia, S., Hogrel, J.-Y., Niks, E.H., de Groot, H., Straub, V., Ricotti, V., Manzur, A., Muntoni, F., Frosini, S., LoMauro, A., Diella, E., Russo, A., Delle Fave, M., Pistininzi, C., Marchi, E., Pascuzzo, R., Vantini, S., Aliverti, A., D’Angelo, M.G., Garibaldi, M., Iacono, S., Di Stefano, V., Lupica, A., Gagliardo, A., Lanza, P., Rispoli, M.G., Ferri, L., Brighina, F., Di Muzio, A., Frangiamore, R., Antozzi, C., Baggi, F., Onore, M.E., Torella, A., Musacchia, F., Del Vecchio Blanco, F., Zanobio, M., Piluso, G., Poggetti, F., Ripolone, M., Zanotti, S., Caputo, V., Megalizzi, D., Bax, C., Ranieri, M., Colantoni, L., Tasca, G., Ricci, E., Caltagirone, C., Cascella, R., Giardina, E., Strafella, C., Lai, E., Marcello, M., Morotti, I., Caremani, M., Conte, I., Linari, M., Raffaghello, L., Principi, E., Baratto, S., Pintus, S., Antonini, F., Del Zotto, G., Bruzzone, S., Scudieri, P., Gazzerro, E., Ronchi, D., Lucchiari, S., Garbellini, M., Salani, S., Ciscato, P., Bresolin, N., Zeuli, R., Varavallo, A., Angelini, C., Brunetti-Pierri, N., Wischmeijer, A., Labella, B., Pezzini, D., Costa, A., Poli, L., Magoni, M., Manini, A., Velardo, D., Cinnante, C.M., Marija, M., Janez, Z., Traverso, M., Pini, A., Giannotta, M., Valentino, M.L., Scala, M., Zara, F., Fiorillo, C., Antognozzi, S., Napoli, L., Scuvera, G., Giacobbe, A., Milani, D., Rolle, E., Rossi, F., Cavallina, I., D’Alessandro, R., Urbano, G., Ricci, F, Peruzzo, D., Ciceri, T., Mascheretti, S., Lampis, V., Arrigoni, F., Giubergia, A., Crippa, A., Nobile, M., Mani, E., Falzarano, M.S., Mietto, M., Rossi, R., Selvatici, R., Gessi, M., Montanaro, F., Morgan, J., Muntoni, F, Lanzi, G., Galvagni, A., Romani, C., Gerevini, S., Selicorni, A., Ajdinaj, P., Montrasio, S., Cazzaniga, S., Bettica, P.U., Mastellaro, S., Romano, F., Napoli, M.M., Barbone, F., De Rosa, M.A., Angelucci, D., Andreassi, G., Amatetti, M., Amerio, P., Altamura, C., Farinato, A., Campanale, C., Carratù, M.R., Desaphy, J.-F., Brugnoni, R., Rossi, T., Canavese, C., Eoli, M., Lauria, G., Toscano, A., DiDuca, M., Campana, C., Cataldi, M., Greco, G., Frezza, E., Goglia, M., Boffa, L., Pignolo, A., Quartana, M., Fierro, B., Musumeci, S.A., Scarsi, E., Cella, A., Narciso, E., Cabona, C., Beronio, A., Assini, A., Del Sette, M., Bandini, F., Benedetti, L., Prada, V., Torre, E., Missaglia, S., Tavian, D., Baldelli, E., Caria, F., Ferrari Aggradi, C.R., Falcier, E., Lizio, A., Pirola, A., Casiraghi, J., Carraro, E., Mauro, L., Rao, F., Roma, E., Iannello, A., De Mattia, E., Barp, A., Lupone, S., Gatti, V., Italiano, C., Frisoni, M.C., Ferrari-Aggradi, C.R., Refran, J., Becchiati, S., Iossa, F., Eichinger, K., Dekdebrun, J., St Romain, J., Johnson, N., Thornton, C., Leali, M., Aimo, A., Todiere, G., Vergaro, G., Grigoratos, C., Giannoni, A., Baldinotti, F., Aquaro, G.D., Emdin, M., Passino, C., Barison, A., Gibertini, S., Iannibelli, E., Mora, M., Ruggieri, A., Scutifero, M., Palladino, A., Spiro Santovito, L., Pasanisi, B., Nesti, C., Bartolini, M., Tarabelloni, A., Tizzoni, F., Colombo, P., Tesei, A., Molteni, M., Falzone, Y., Chico, L., Caligo, M.A., D’Apice, M.R., Mantegazza, R.E., Pegolo, E., Cibin, F., Sawacha, Z., Giagnorio, E., Malacarne, C., Salvi, E., Bossolasco, P., Bardelli, D., Ratti, A., Marcuzzo, S., Melzi, V., Rizzuti, M., Gagliardi, D., Masrori, P., Biella, F., Van Damme, P., Musso, G., Cosma, C., Plebani, M., Neri, M., Sette, E., Trabanelli, C., Margutti, A., Rimessi, P., Fabris, M., Tugnoli, V., Gualandi, F., Paoletti, M., Diamanti, L., Muzic, S.I., Ballante, E., Solazzo, F., Foppoli, L., Deligianni, X., Santini, S., Figini, S., Bergsland, N., Pichiecchio, A., Pilla, F., Ianes, P.G., Casagrande, S., Gasperini, B., Zuccarino, R., Bersani, M., Pagliari, E., Saccomanno, D., Cattaneo, C., Lops, J., Pera, M.C., Palazzo, G., Scarpini, G., Not, R., Capelli, P., Testoni, C., Pisani, F., Piccolo, B., Fusco, C., Frattini, D., Vergine, G., Farina, M., Sarajlia, J., Marchetti, F., Piccinini, G.
Publikováno v:
Acta Myologica
Background Risdiplam has been approved for Compassionate Use (CUP) since 2019, for SMA type 1 and type 2 who could not receive the approved treatment for the disease. It’s an orally administered drug targeting SMN2 gene. More than 200 patients have
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmc_________::cc20b5f2e1c8b4b118a3c86c48d1857d
http://europepmc.org/articles/PMC8694696
http://europepmc.org/articles/PMC8694696
Autor:
Primiano G; Dipartimento di Neuroscienze, Organi di Senso e Torace, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Dipartimento Di Neuroscienze, Università Cattolica del Sacro Cuore, Rome, Italy., Plantone D; Department of Medicine, Surgery and Neuroscience, University of Siena, Siena, Italy., Piro G; Department of Medical and Surgical Sciences, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Carbone C; Department of Medical and Surgical Sciences, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Sabino A; Dipartimento Di Neuroscienze, Università Cattolica del Sacro Cuore, Rome, Italy., Sancricca C; Dipartimento di Neuroscienze, Organi di Senso e Torace, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Fondazione UILDM Lazio Onlus, Rome, Italy., Di Nottia M; Unit of Cellular Biology and Mitochondrial Disease, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Carrozzo R; Unit of Cellular Biology and Mitochondrial Disease, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Servidei S; Dipartimento di Neuroscienze, Organi di Senso e Torace, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Dipartimento Di Neuroscienze, Università Cattolica del Sacro Cuore, Rome, Italy.
Publikováno v:
European journal of neurology [Eur J Neurol] 2023 Oct; Vol. 30 (10), pp. 3409-3410. Date of Electronic Publication: 2023 Jul 16.
Autor:
Astrea, G., Battini, R., Berardinelli, A., Bertini, E., Bruno, C., Catteruccia, M., Comi, G.P., D’Amico, A., Fattori, F., Fiorillo, C., Magri, F., Mercuri, E., Messina, S., Mongini, T., Mora, M., Morani, F., Moro, F., Pane, M., Pegoraro, E., Pini, A., Politano, L., Ricci, F., Sframeli, M., Toscano, A., Santorelli, F.M., Lenzi, S., Bello, L., Corti, S., Donati, M.A., Tubili, F., Morrone, A., la Marca, G., Daniotti, M., Evoli, A., Ferlini, A., Garibaldi, M., Rendu, J., Brocard, J., Lacene, E., Fauré, J., Brochier, G., Beuvin, M., Labasse, C., Madelaine, A., Malfatti, E., Bevilacqua, J.A., Lubieniecki, F., Monges, S., Taratuto, A.L., Laporte, J., Marty, I., Antonini, G., Romero, N.B., M.L.*, Lombardo, Maggi, L., Mirabella, M., Musumeci, O., Paoletti, M., Pichiecchio, A., Pennisi, E.M., Ricci, G., Rodolico, C., Marchese, M., Siciliano, G., Tasca, G., Mele, F., Ruggiero, L., Vercelli, L., Bettio, C., Tripodi, S., Govi, M., Bucci, E., Di Muzio, A., Scarlato, M., Villa, L., D’Angelo, G., Filosto, M., Piras, R., Maioli, M.A., Massa, R., Previtali, S., Angelini, C., Moggio, M., Santoro, L., Tomelleri, G., Tupler, R., Udd, B., Sera, F., Maranda, L., Vianello, A., Vissing, J., Zeviani, M., Barp, A., Laforet, P., Monforte, M., Ricci, E., Choumert, A., Stojkovic, T., Semplicini, C., Stramare, R., Scheidegger, O., Haberlova, J., Straub, V., Marini Bettolo, C., Løkken, N., Diaz-Manera, J., Urtizberea, J.A., Kynčl, M., Walter, M.C., Carlier, R.Y., Zambon, A.A., Albamonte, E., Baranello, G., Gandossini, S., Gualandi, F., Natali Sora, M.G., Politano, A., Sansone, V., Vita, G.L., Previtali, S.C., Villa, M., Fusto, A., Vianello, S., Merlo, B., Pedemonte, M., Tacchetti, P., Lanzillotta, V., Trucco, F., De Mattia, E., Rao, F., Calore, C., Hoffman, E.P., Morgenroth, L., Gordish-Dressman, H., McDonald, C.M., Costa, R., Rodia, M.T., Santi, S., Lattanzi, G., Papa, V., Pegoraro, V., Cenacchi, G., M, Sframeli, Ciranni, A., Versaci, A., Di Bella, V., Ferlazzo, V., Gitto, E., Aguennouz, M., Vita, G., Mendell, J.R., Al-Zaidy, S., Lehman, K.J., McColly, M., Lowes, L.P., Alfano, L.N., Miller, N.F., Iammarino, M.A., Church, K., Bernat Fuertes, M., Ogrinc, F.G., L’Italien, J., Kernbauer, E., Shah, S., Sproule, D.M., Feltner, D.E., Day, J.W., Chiriboga, C.A., Crawford, T.O., Darras, B.T., Finkel, R.S., Connolly, A.M., Iannaccone, S.T., Kuntz, N.L., Peña, L.D.M., Schultz, M., Shieh, P.B., Smith, E.C., Shah, A., Ouyang, H., Macek, T.A., Muehring, L.M., Kaspar, B.K., Masson, R., Servais, L., Deconinck, N., Klein, A., Darras, B., Kletzl, H., Cleary, H., El-Khairi, M., Seabrook, T., Czech, C., Gerber, M., Nguyen, C., Gelblin, K., Gorni, K., Rossi, R., Falzarano, M.F., Margutti, A., Spedicato, N., El Dani, R., Fabris, M., Neri, M., Fini, S., Rimessi, P., Johansson, C., Al-Khalili Szigyarto, C., Savarese, M., Johari, M., Jonson, P.H., Koivunen, S., Quareshi, T., Vihola, A., Hackman, P., Gemelli, C., Trevisan, L., Fabbri, S., Pisciotta, L., Meo, G., Traverso, M., Zara, F., Minetti, C., Schenone, A., Mandich, P., Grandis, M., C, Dosi, Cassandrini, D., Rubegni, A., Tolomeo, D., Giannini, F., Malandrini, A., Tonin, P., Volpi, N., Potter, R.A., Griffin, D.A., Heller, K.N., Rodino-Klapac, L.R., Salsi, V., Salani, M., Kaufman, P.D., Green, M.R., Vallarola, A., Termanini, A., Cortini, M., Ghiaroni, V., Forcato, M., Germinario, E., D’Antona, G., Blaauw, B., Genazzani, A.A., Filigheddu, N., Santoro, M., Cotti Piccinelli, S., Lamperti, C., Servidei, S., Santorelli, F.A., Simoncini, C., Primiano, G., Galvagni, A., Caria, F., Gallo Cassarino, S., Baldelli, E., Necchini, N., Carelli, V., Padovani, A., Mancuso, M., Verardo, M., Lupica, A., Ripolone, M., Vattemi, G., Sciacco, M., Nigro, V., Lucchini, M., De Arcangelis, V., De Fino, C., Santovito, L., Brigati, G., Diana, C., Panicucci, C., Broda, P., Nesti, C., Santorelli, F., Baronchelli, C., G, Greco, Frezza, E., Rastelli, E., Terracciano, C., Merlonghi, G., Pugliese, S., Tartaglione, T., Calabrò, F., Petrucci, A., M, Catteruccia, Colia, G., Bonetti, A.M., Carlesi, A., Bruno, G.M., Di Matteo, S., Valentino, M.C., Oselin, M., Martinotti, C., Xoxi, E., Colombo, G.L., Russo, A., LoMauro, A., Velardo, D., Turconi, A.C., Bresolin, N., Aliverti, A., D’Angelo, M.G., Ferrero, A., Rossi, M., Palermo, C., Giannotta, M., Rolle, E., Derchi, M., Gardani, A., Balottin, U., Giugliano, T., Torella, A., Garofalo, A., Onore, M.E., Del Vecchio Blanco, F., Piluso, G., Selvatici, R., Trabanelli, C., Buldrini, B., Venturoli, A., Fortunato, F., Potulska, A., Emandi, A.C., Lehman, I., Herczegfalvi, A., Guergueltcheva, V., Kyriakides, T., Sifi, Y., Molnar, M.J., Burnyte, B., Shatillo, A., Vlodavets, D., F.1, Gualandi, Scali, M., Ciscato, P., Menni, F., Mani, E., Falzarano, M.S., Baratto, S., Nesich, V., Iacomino, M., Castiglione, V., Giannoni, A., Florio, F., Rocchi, A., Emdin, M., Fratazzi, C., Naughton, E., Krenz, H., Distefano, M.G., Madia, F., Mauri, E., Ronchi, D., Govoni, A., Brusa, R., Zangaro, V., Lazzarotto, A., Fanin, M., Picillo, E., Ergoli, M., Principi, E., Del Zotto, G., Antonini, F., Ognio, M., Bruzzone, S., Gazzerro, E., Raffaghello, L., Lai, E., Torri, F., Chico, L., Fuccillo, E., Nucera, G., Greco, G., Di Mauro, R., Di Girolamo, S., Siliquini, S., Meneri, M., Cinnante, C., Triulzi, F., Vergari, M., Cogiamanian, F., Stocchetti, N., Calderini, E., Sansone, V.A., Corti, S.P., Pera, M.C., Kirschner, J., Goemans, N., Tichy, M., Yeung, W.Y., Annoussamy, M., Cleary, Y., Carraro, E., Salmin, F., Lunetta, C., Comi, G., Sconfienza, L.M., Manenti, G.F., Valeria, V., Morettini, V., Gagliano, N., Bonanno, S., Marcuzzo, S., Malacarne, C., Giagnorio, E., Zanin, R., Andreetta, F., Simoncini, O., Bernasconi, P., Mantegazza, R., Cherchi, C., Chiarini Testa, M.B., Bonetti, A., Rollo, M., Bianchi, R., Longo, A., Nicita, F., Corsetti, T., Cutrera, R., Bruno, G., Allegorico, L., Lombardi, L., Napolitano, F., Sampaolo, S., Bertocci, G., Guglielmi, V., Matà, S., Federico, A., Merlini, L., Matucci-Cerinic, C., Fionda, L., Vanoli, V., Leonardi, L., Loreti, S., Morino, S., Micaloni, A., Raffa, S., Blasio, G., Varone, A., Marrosu, G., Moroni, I., Fusco, C., Sabatelli, P., Morandi, L., Grilli, A., Bicciato, S., Palmio, J., Poza, J.J., Weinberg, J., Olive, M., Cobo, A.M., Sarparanta, J., García-Bragado, F., Gibertini, S., Saredi, S., Matalonga, L., Farina, L., Ardissone, A., Caumo, L., Bozzoni, V., Cester, G., Gabrieli, J., Causin, F., Sorarù, G., Galatolo, D., D’Amore, F., Doccini, S., Giglio, S., Pantaleo, M., Guarducci, S., Telese, R., De Rosa, A., Napoli, L., Fagiolari, G., Montomoli, M., Mancardi, M.M., Mari, F., Morana, G., Guerrini, R., Arceri, S., Ravaglia, S., Cosentino, G., Alfonsi, E., Pugliese, A., Volta, S., Oteri, R., Della Marca, G., Brunetti, V., Sancricca, C., Vollono, C., Macone, A., Missaglia, S., Noguera, N.I., Palma, E., Tavian, D., Pennisi, E.M.M., Tavilla, G., DiSfetano, M.G., Di Iorio, G., Melone, M.A.B., Esposito, T., Tartara, A., Cena, H., Stefan, C., Brondani, G., Trevisi, E., Martinuzzi, A., Brizzi, T., Peverelli, L., Legati, A., Lamantea, E., Nasca, A., Marchet, S., Lerario, A., Galimberti, V., Ghezzi, D., Vitale, R., Antonio Toscano, A., Sacchini, M., D’Angelo, R., Boschetti, E., Caporali, L., Lodi, R., Pironi, L., De Giorgio, R., Rinaldi, R., Fernandez-Vizarra, E., Tiranti, V., Pinzan, E., Marozzo, R., Montano, V., Calì Cassi, L., Botta, A., Silvestri, G., Cintoni, M., Pulcini, G., Palombi, C., Salvia, M., Tammam, G., De Lorenzo, A., Carlesi, D., Diodato, D., Bonanno, C., Nicocia, G., Foti, F.M., Ricciardi, R., Bocci, T., Maestri, M., Guida, M., Bonuccelli, U., Vanoli, F., Di Pasquale, A., Vizzaccaro, E., Sinicropi, S., Pancheri, E., Sajeva, G., Goffi, F., Zanoni, M., Bertolasi, L., Abati, E., Girolamo, F., Lia, A., Giannini, M., Fornaro, M., Amati, A., D’Abbicco, D., Tampoia, M., Serlenga, L., Iannone, F., Trojano, M., Frigeri, A., Ticci, C, Battisti, C., Melani, F., Alì, G., Sartucci, F., Ceppa, I., Dosi, C., Baldacci, J., Dati, E., Frosini, S., Conte, R., Calderisi, M., Sansone, F., Diodato, G., Pala, A.P., Scudellari, M.C., Tonacci, A., Ceppa, C., Giorgolo, F., Marfisi, D., Del Bo, R., Faravelli, I., Gagliardi, D., Costamagna, G., Torrente, Y., Malcontenti, S., Coluccini, M., Perazza, S., Amador, C., Grande, A., Roccella, S., Spezzaneve, A., Fantacci, M.E., Iori, E., Ariatti, A., Mazzoli, M., Fini, N., Genovese, M., Galassi, G., Sette, E., Tugnoli, V., Rispoli, M.G., Mero, S., Vitale, M., Barbone, F., Di Stefano, V., De Angelis, M.V., Ruggieri, A., Cauley, E.S., Spivey, T., Manzini, M.C., Orlandi, R., Gajofatto, A.
Publikováno v:
Acta Myologica
GMPPB, encoding the guanosine-diphosphate-mannose (GDP-mannose) pyrophosphorylase B protein, has recently been associated with a wide clinical spectrum ranging from severe Walker-Warburg syndrome to pseudo-metabolic myopathy and even congenital myast
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid________::0a1599d89e0cd061ff2824e8b2eaabff
http://europepmc.org/articles/PMC6598409
http://europepmc.org/articles/PMC6598409
Autor:
Lombardo, M. E., Carraro, E., Sancricca, C., Armando, M., Catteruccia, M., Mazzone, E., Ricci, G., Salamino, F., Santorelli, F. M., Filosto, M.
Publikováno v:
Acta Myol
Muscular dystrophy (MD) is a group of neuromuscular diseases characterized by progressive muscle weakness due to various mutations in several genes involved in muscle structure and function. The age at onset, evolution and severity of the different f
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid_dedup__::d6a99f03a246b9546078f5a491f7b1f8
https://europepmc.org/articles/PMC8290512/
https://europepmc.org/articles/PMC8290512/
Autor:
Fortunato F; Medical Genetics Unit, Department of Medical Sciences, University of Ferrara, Ferrara, Italy.; Medical Genetics Unit, Department of Mother and Child, Sant'Anna University Hospital of Ferrara, Ferrara, Italy., Bianchi F; Department of Clinical and Experimental Medicine, Neurological Clinic, University of Pisa, Pisa, Italy., Ricci G; Department of Clinical and Experimental Medicine, Neurological Clinic, University of Pisa, Pisa, Italy., Torri F; Department of Clinical and Experimental Medicine, Neurological Clinic, University of Pisa, Pisa, Italy., Gualandi F; Medical Genetics Unit, Department of Mother and Child, Sant'Anna University Hospital of Ferrara, Ferrara, Italy., Neri M; Medical Genetics Unit, Department of Mother and Child, Sant'Anna University Hospital of Ferrara, Ferrara, Italy., Farnè M; Medical Genetics Unit, Department of Medical Sciences, University of Ferrara, Ferrara, Italy.; Medical Genetics Unit, Department of Mother and Child, Sant'Anna University Hospital of Ferrara, Ferrara, Italy., Giannini F; Department of Medical, Surgical and Neurological Sciences, University of Siena, Siena, Italy., Malandrini A; Department of Medical, Surgical and Neurological Sciences, University of Siena, Siena, Italy., Volpi N; Department of Medical, Surgical and Neurological Sciences, University of Siena, Siena, Italy., Lopergolo D; Department of Medical, Surgical and Neurological Sciences, University of Siena, Siena, Italy., Silani V; Department of Neurology and Laboratory of Neuroscience, IRCCS Istituto Auxologico Italiano, Milan, Italy.; Department of Pathophysiology and Transplantation, 'Dino Ferrari' Center, Università Degli Studi Di Milano, Milan, Italy., Ticozzi N; Department of Neurology and Laboratory of Neuroscience, IRCCS Istituto Auxologico Italiano, Milan, Italy.; Department of Pathophysiology and Transplantation, 'Dino Ferrari' Center, Università Degli Studi Di Milano, Milan, Italy., Verde F; Department of Neurology and Laboratory of Neuroscience, IRCCS Istituto Auxologico Italiano, Milan, Italy.; Department of Pathophysiology and Transplantation, 'Dino Ferrari' Center, Università Degli Studi Di Milano, Milan, Italy., Pareyson D; Unit of Rare Neurodegenerative and Neurometabolic Diseases, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy., Fenu S; Unit of Rare Neurodegenerative and Neurometabolic Diseases, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy., Bonanno S; Neuroimmunology and Neuromuscular Diseases Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy., Nigro V; Department of Precision Medicine, University of Campania 'L. Vanvitelli', Naples, Italy.; Telethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Naples, Italy., Peduto C; Department of Precision Medicine, University of Campania 'L. Vanvitelli', Naples, Italy., D'Ambrosio P; Department of Precision Medicine, University of Campania 'L. Vanvitelli', Naples, Italy., Zeuli R; Department of Precision Medicine, University of Campania 'L. Vanvitelli', Naples, Italy., Zanobio M; Department of Precision Medicine, University of Campania 'L. Vanvitelli', Naples, Italy., Picillo E; Department of Precision Medicine, University of Campania 'L. Vanvitelli', Naples, Italy., Servidei S; Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Primiano G; Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Sancricca C; Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Sciacco M; Neuromuscular and Rare Disease Unit, Department of Neuroscience, Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy., Brusa R; Neuromuscular and Rare Disease Unit, Department of Neuroscience, Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy., Filosto M; Department of Clinical and Experimental Sciences, University of Brescia, Brescia, Italy.; ASST Spedali Civili Di Brescia, Brescia, Italy.; NeMO-Brescia Clinical Center for Neuromuscular Diseases, Brescia, Italy., Cotti Piccinelli S; Department of Clinical and Experimental Sciences, University of Brescia, Brescia, Italy.; ASST Spedali Civili Di Brescia, Brescia, Italy.; NeMO-Brescia Clinical Center for Neuromuscular Diseases, Brescia, Italy., Pegoraro E; Department of Neuroscience, University of Padova, Padua, Italy., Mongini T; Department of Neurosciences 'Rita Levi Montalcini', University of Turin, Turin, Italy., Solero L; Department of Neurosciences 'Rita Levi Montalcini', University of Turin, Turin, Italy., Gadaleta G; Department of Neurosciences 'Rita Levi Montalcini', University of Turin, Turin, Italy., Brusa C; Department of Neurosciences 'Rita Levi Montalcini', University of Turin, Turin, Italy., Minetti C; Pediatric Neurology Unit and Muscle Unit, IRCCS Istituto Giannina Gaslini, Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genoa, Genoa, Italy., Bruno C; Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genoa, Genoa, Italy., Panicucci C; Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genoa, Genoa, Italy., Sansone VA; The NEMO (NEuroMuscular Omniservice) Clinical Center, Milan, Italy.; Neurorehabilitation Unit, University of Milan, Milan, Italy., Lunetta C; The NEMO (NEuroMuscular Omniservice) Clinical Center, Milan, Italy., Zanolini A; The NEMO (NEuroMuscular Omniservice) Clinical Center, Milan, Italy., Toscano A; Neurology and Neuromuscular Diseases Unit, Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.; ERN-NMD Center of Messina, Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy., Pugliese A; Neurology and Neuromuscular Diseases Unit, Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy., Nicocia G; Neurology and Neuromuscular Diseases Unit, Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy., Bertini E; Unit of Neuromuscular and Neurodegenerative Disorders, Department of Neurosciences, Bambino Gesù Children's Hospital IRCCS, Rome, Italy., Catteruccia M; Unit of Neuromuscular and Neurodegenerative Disorders, Department of Neurosciences, Bambino Gesù Children's Hospital IRCCS, Rome, Italy., Diodato D; Unit of Neuromuscular and Neurodegenerative Disorders, Department of Neurosciences, Bambino Gesù Children's Hospital IRCCS, Rome, Italy., Atalaia A; Service of Neuromyology, APHP-GH Pitié-Salpêtrière, Sorbonne Université, Paris, France., Evangelista T; Neuromuscular Morphology Unit, Institute of Myology, GHU Pitié-Salpêtrière, Sorbonne Université, Paris, France., Siciliano G; Department of Clinical and Experimental Medicine, Neurological Clinic, University of Pisa, Pisa, Italy., Ferlini A; Medical Genetics Unit, Department of Medical Sciences, University of Ferrara, Ferrara, Italy. fla@unife.it.; Medical Genetics Unit, Department of Mother and Child, Sant'Anna University Hospital of Ferrara, Ferrara, Italy. fla@unife.it.
Publikováno v:
Orphanet journal of rare diseases [Orphanet J Rare Dis] 2023 Jul 21; Vol. 18 (1), pp. 196. Date of Electronic Publication: 2023 Jul 21.