Clinical, Histopathologic, and Genetic Features of Patients With Myofibrillary and Distal Myopathies: Experience From the Italian Network.

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.