Zobrazeno 1 - 10
of 982
pro vyhledávání: '"Sansone VA"'
Publikováno v:
Journal of Clinical Sleep Medicine. 17:2351-2352
Autor:
Pane M; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.; Centro Clinico Nemo, Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Stanca G; Centro Clinico Nemo, Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Coratti G; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.; Centro Clinico Nemo, Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., D' Amico A; Unit of Muscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Sansone VA; The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy., Berti B; Centro Clinico Nemo, Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Fanelli L; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.; Centro Clinico Nemo, Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Albamonte E; The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy., Ausili Cefaro C; Speech Language Pathology Unit, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Lazio, Italy., Cerchiari A; Feeding and Swallowing Services Unit, Bambino Gesù Children's Hospital IRCCS, Rome, Italy., Catteruccia M; Unit of Muscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., De Sanctis R; Centro Clinico Nemo, Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Leone D; Centro Clinico Nemo, Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Palermo C; Centro Clinico Nemo, Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Buchignani B; Centro Clinico Nemo, Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Department of Translational Research and of New Surgical and Medical Technologies, University of Pisa, Pisa, Italy., Onesimo R; Center for Rare Diseases and Birth Defects, Department of Woman and Child Health and Public Health, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy., Kuczynska EM; Center for Rare Diseases and Birth Defects, Department of Woman and Child Health and Public Health, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy., Tosi M; Unit of Muscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Pera MC; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.; Centro Clinico Nemo, Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Bravetti C; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.; Centro Clinico Nemo, Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Tiziano FD; Department of Life Sciences and Public Health, Section of Genomic Medicine, Università Cattolica del Sacro Cuore, Rome, Italy., Bertini E; Unit of Muscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Mercuri E; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy. eugeniomaria.mercuri@policlinicogemelli.it.; Centro Clinico Nemo, Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy. eugeniomaria.mercuri@policlinicogemelli.it.
Publikováno v:
European journal of pediatrics [Eur J Pediatr] 2024 Aug 29. Date of Electronic Publication: 2024 Aug 29.
Autor:
Panicucci C; Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Via G. Gaslini, 5, I-16147, Genova, Italy., Sahin E; Department of Biostatistics and Bioinformatics, Institute of Health Sciences, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey., Bartolucci M; Core Facilities-Clinical Proteomics and Metabolomics, IRCCS Istituto Giannina Gaslini, Genova, Italy., Casalini S; Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Via G. Gaslini, 5, I-16147, Genova, Italy., Brolatti N; Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Via G. Gaslini, 5, I-16147, Genova, Italy., Pedemonte M; Pediatric Neurology Unit, IRCCS Istituto Giannina Gaslini, Genova, Italy., Baratto S; Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Via G. Gaslini, 5, I-16147, Genova, Italy., Pintus S; Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Via G. Gaslini, 5, I-16147, Genova, Italy., Principi E; Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Via G. Gaslini, 5, I-16147, Genova, Italy., D'Amico A; Unit of Neuromuscular and Neurodegenerative Disorders, IRCCS Bambino Gesù Children's Hospital, Rome, Italy., Pane M; Centro Clinico Nemo, IRCCS Fondazione Policlinico Universitario Agostino Gemelli, Rome, Italy., Sframeli M; Department of Neurosciences, University of Messina, Messina, Italy., Messina S; Department of Neurosciences, University of Messina, Messina, Italy., Albamonte E; Neurorehabilitation Unit, Centro Clinico NeMO, University of Milan, Milan, Italy., Sansone VA; Neurorehabilitation Unit, Centro Clinico NeMO, University of Milan, Milan, Italy., Mercuri E; Centro Clinico Nemo, IRCCS Fondazione Policlinico Universitario Agostino Gemelli, Rome, Italy., Bertini E; Unit of Neuromuscular and Neurodegenerative Disorders, IRCCS Bambino Gesù Children's Hospital, Rome, Italy., Sezerman U; Department of Biostatistics and Medical Informatics, School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey., Petretto A; Core Facilities-Clinical Proteomics and Metabolomics, IRCCS Istituto Giannina Gaslini, Genova, Italy., Bruno C; Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Via G. Gaslini, 5, I-16147, Genova, Italy. claudiobruno@gaslini.org.; Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health- DINOGMI, University of Genova, Genova, Italy. claudiobruno@gaslini.org.
Publikováno v:
Cellular and molecular life sciences : CMLS [Cell Mol Life Sci] 2024 Sep 10; Vol. 81 (1), pp. 393. Date of Electronic Publication: 2024 Sep 10.
Autor:
Trucco F; NeMO Clinical Center, Fondazione Serena, Milan, Italy.; Department of Neurorehabilitation, University of Milan, Milan, Italy.; Paediatric Neurology and Muscular Diseases Unit, Istituto di Ricovero e Cura a Carattere Scientifico Istituto Giannina Gaslini, Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genova, Genoa, Italy., Albamonte E; NeMO Clinical Center, Fondazione Serena, Milan, Italy., Pane M; Centro Clinico NeMO, Fondazione Policlinico Universitario Agostino Gemelli Istituto di Ricovero e Cura a Carattere Scientifico, Rome, Italy.; Paediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy., Ricci F; Department of Sciences of Public Health and Pediatrics, University of Turin, Turin, Italy., D'amico A; Unit of Muscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, Istituto di Ricovero e Cura a Carattere Scientifico, Rome, Italy., Astrea G; Department of Developmental Neuroscience, Istituto di Ricovero e Cura a Carattere Scientifico Fondazione Stella Maris, Pisa, Italy., Moroni I; Department of Pediatric Neurosciences, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Istituto Neurologico Carlo Besta, Milan, Italy., Pini A; Pediatric Neuromuscular Unit, UOC Neuropsichiatria Dell'Età Pediatrica, Istituto di Ricovero e Cura a Carattere Scientifico Istituto delle Scienze Neurologiche di Bologna, Bologna, Italy., Fiorillo C; Unit of Child Neuropsychiatry, Istituto di Ricovero e Cura a Carattere Scientifico Istituto Giannina Gaslini and DINOGMI, University of Genova, Genoa, Italy., Berardinelli A; Child and Adolescent Neuromuscular Disorder Unit, Istituto di Ricovero e Cura a Carattere Scientifico Mondino Foundation, Pavia, Italy., Johnson NE; Department of Neurology, Virginia Commonwealth University, Richmond, VA, USA., Sansone VA; NeMO Clinical Center, Fondazione Serena, Milan, Italy.; Department of Neurorehabilitation, University of Milan, Milan, Italy.
Publikováno v:
Developmental medicine and child neurology [Dev Med Child Neurol] 2024 Sep 04. Date of Electronic Publication: 2024 Sep 04.
Autor:
Scarano S; Department of Biomedical Sciences for Health, University of Milan, 20133 Milan, Italy.; IRCCS Istituto Auxologico Italiano, Department of Neurorehabilitation Sciences, Ospedale San Luca, 20122 Milan, Italy., Caronni A; Department of Biomedical Sciences for Health, University of Milan, 20133 Milan, Italy.; IRCCS Istituto Auxologico Italiano, Department of Neurorehabilitation Sciences, Ospedale San Luca, 20122 Milan, Italy., Carraro E; The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan, 20162 Milan, Italy., Ferrari Aggradi CR; The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan, 20162 Milan, Italy., Rota V; IRCCS Istituto Auxologico Italiano, Department of Neurorehabilitation Sciences, Ospedale San Luca, 20122 Milan, Italy., Malloggi C; IRCCS Istituto Auxologico Italiano, Department of Neurorehabilitation Sciences, Ospedale San Luca, 20122 Milan, Italy., Tesio L; IRCCS Istituto Auxologico Italiano, Department of Neurorehabilitation Sciences, Ospedale San Luca, 20122 Milan, Italy., Sansone VA; Department of Biomedical Sciences for Health, University of Milan, 20133 Milan, Italy.; The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan, 20162 Milan, Italy.
Publikováno v:
Journal of clinical medicine [J Clin Med] 2024 Aug 09; Vol. 13 (16). Date of Electronic Publication: 2024 Aug 09.
Autor:
Coratti G; Pediatric Neurology Unit, Catholic University, 00135 Rome, Italy; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Rome, Italy., Civitello M; St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Rohwer A; Department of Developmental Neuroscience, Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK; Department of Neurology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Salmin F; The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, 20162 Milan, Italy., Glanzman AM; Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Montes J; Columbia University Irving Medical Center, New York, NY 10032, USA., Pasternak A; Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA., De Sanctis R; Pediatric Neurology Unit, Catholic University, 00135 Rome, Italy; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Rome, Italy., Young SD; Departments of Neurology TD, Stanford University, Palo Alto, CA 94305, USA., Duong T; Departments of Neurology TD, Stanford University, Palo Alto, CA 94305, USA., Mizzoni I; Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Medical Genetics, Bambino Gesù Children's Hospital, IRCCS, 00165 Rome, Italy., Milev E; Department of Developmental Neuroscience, Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK; Department of Neurology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Sframeli M; Department of Clinical and Experimental Medicine, University of Messina, 98122 Messina, Italy., Morando S; Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophthalmology Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini and University of Genoa, 16132 Genoa, Italy., Albamonte E; The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, 20162 Milan, Italy., D'Amico A; Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Medical Genetics, Bambino Gesù Children's Hospital, IRCCS, 00165 Rome, Italy., Brolatti N; Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophthalmology Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini and University of Genoa, 16132 Genoa, Italy., Pane M; Pediatric Neurology Unit, Catholic University, 00135 Rome, Italy; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Rome, Italy., Scoto M; Department of Developmental Neuroscience, Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK; Department of Neurology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Messina S; Department of Clinical and Experimental Medicine, University of Messina, 98122 Messina, Italy., Hirano M; Columbia University Irving Medical Center, New York, NY 10032, USA., Zolkipli-Cunningham Z; Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; University of Pennsylvania, Philadelphia, PA 19104, USA., Darras BT; Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA., Bertini E; Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Medical Genetics, Bambino Gesù Children's Hospital, IRCCS, 00165 Rome, Italy., Bruno C; Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophthalmology Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini and University of Genoa, 16132 Genoa, Italy., Sansone VA; The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, 20162 Milan, Italy., Day J; Departments of Neurology TD, Stanford University, Palo Alto, CA 94305, USA., Baranello G; Department of Developmental Neuroscience, Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK; Department of Neurology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Pera MC; Pediatric Neurology Unit, Catholic University, 00135 Rome, Italy; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Rome, Italy., Muntoni F; Department of Neurology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK; School of Health and Sports Sciences, University of Suffolk, Ipswich IP4 1QJ, UK., Finkel R; St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Mercuri E; Pediatric Neurology Unit, Catholic University, 00135 Rome, Italy; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Rome, Italy. Electronic address: eugeniomaria.mercuri@unicatt.it.
Publikováno v:
Neuromuscular disorders : NMD [Neuromuscul Disord] 2024 Aug; Vol. 41, pp. 42-50. Date of Electronic Publication: 2024 May 09.
Autor:
Coratti G; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Bovis F; Department of Health Sciences (DISSAL), University of Genoa, Genoa, Italy., Pera MC; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Scoto M; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health & MRC Centre for Neuromuscular Diseases, London, UK., Montes J; Columbia University Irving Medical Center, New York, New York, USA., Pasternak A; Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA., Mayhew A; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle Upon Tyne, UK., Muni-Lofra R; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle Upon Tyne, UK., Duong T; Department of Neurology TD, Stanford University, Palo Alto, California, USA., Rohwer A; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health & MRC Centre for Neuromuscular Diseases, London, UK., Dunaway Young S; Department of Neurology TD, Stanford University, Palo Alto, California, USA., Civitello M; St. Jude Children's Research Hospital, Memphis, Tennessee, USA., Salmin F; NEMO Clinical Center, Milan, Italy., Mizzoni I; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital IRCCS, Rome, Italy., Morando S; Center of Myology and Neurodegenerative Disorders, IRCCS Istituto Giannina Gaslini, Genoa, Italy., Pane M; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Albamonte E; NEMO Clinical Center, Milan, Italy., D'Amico A; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital IRCCS, Rome, Italy., Brolatti N; Center of Myology and Neurodegenerative Disorders, IRCCS Istituto Giannina Gaslini, Genoa, Italy., Sframeli M; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy., Marini-Bettolo C; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle Upon Tyne, UK., Sansone VA; NEMO Clinical Center, Milan, Italy.; Department of Biomedical Sciences for Health, University of Milan, Milan, Italy., Bruno C; Center of Myology and Neurodegenerative Disorders, IRCCS Istituto Giannina Gaslini, Genoa, Italy., Messina S; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy., Bertini E; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital IRCCS, Rome, Italy., Baranello G; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health & MRC Centre for Neuromuscular Diseases, London, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK., Day J; Department of Neurology TD, Stanford University, Palo Alto, California, USA., Darras BT; Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA., De Vivo DC; Columbia University Irving Medical Center, New York, New York, USA., Hirano M; Columbia University Irving Medical Center, New York, New York, USA., Muntoni F; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health & MRC Centre for Neuromuscular Diseases, London, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK., Finkel R; St. Jude Children's Research Hospital, Memphis, Tennessee, USA., Mercuri E; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
Publikováno v:
European journal of neurology [Eur J Neurol] 2024 Aug; Vol. 31 (8), pp. e16309. Date of Electronic Publication: 2024 Apr 24.
Autor:
Lizio A; The NEMO Clinical Center (NEuroMuscular Omnicenter - Fondazione Serena Onlus), Milan, Italy., Greco L; The NEMO Clinical Center (NEuroMuscular Omnicenter - Fondazione Serena Onlus), Milan, Italy.; NeMO Lab, ASST Niguarda Cà Granda Hospital, Milan, Italy., Beretta M; The NEMO Clinical Center (NEuroMuscular Omnicenter - Fondazione Serena Onlus), Milan, Italy., Frisoni MC; The NEMO Clinical Center (NEuroMuscular Omnicenter - Fondazione Serena Onlus), Milan, Italy., Becchiati S; The NEMO Clinical Center (NEuroMuscular Omnicenter - Fondazione Serena Onlus), Milan, Italy., Casiraghi J; The NEMO Clinical Center (NEuroMuscular Omnicenter - Fondazione Serena Onlus), Milan, Italy., Sansone VA; The NEMO Clinical Center (NEuroMuscular Omnicenter - Fondazione Serena Onlus), Milan, Italy.; Neurorehabilitation Unit, University of Milan, Milan, Italy., Carraro E; The NEMO Clinical Center (NEuroMuscular Omnicenter - Fondazione Serena Onlus), Milan, Italy.
Publikováno v:
Disability and rehabilitation [Disabil Rehabil] 2024 Jul; Vol. 46 (15), pp. 3462-3468. Date of Electronic Publication: 2023 Aug 14.
Autor:
Trucco F; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Ridout D; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Weststrate H; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Scoto M; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Rohwer A; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Coratti G; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Main ML; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Mayhew AG; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Montes J; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., De Sanctis R; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Pane M; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Pera MC; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Sansone VA; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Albamonte E; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., D'Amico A; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Bruno C; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Messina SS; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Childs AM; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Willis T; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Ong MT; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Servais L; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Majumdar A; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Hughes I; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Marini-Bettolo C; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Parasuraman D; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Gowda VL; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Baranello G; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Bertini ES; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., De Vivo DC; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Darras BT; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Day JW; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Mayer O; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Zolkipli-Cunningham Z; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Finkel RS; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Mercuri E; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom., Muntoni F; Dubowitz Neuromuscular Centre (FT, HW, MS, AR, MLM, GB, FM), UCL Institute of Child Health & Great Ormond Street Hospital, London; Department Paediatric Neuroscience Evelina London Children's Hospital and Department Paediatric Respiratory Medicine (FT), Royal Brompton Hospital, Guy's and St Thomas NHS Trust London, United Kingdom; Pediatric Neurology and Muscular Diseases Unit (FT), IRCCS Istituto Giannina Gaslini, Genova and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Italy; Population (DR), Policy and Practice Programme, UCL GOS Institute of Child Health, London; Paediatric Neurology (GC, RDS, MP, MCP, EM), Università Cattolica del Sacro Cuore; Centro Clinico Nemo (GC, RDS, MP, MCP, EM), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Newcastle University and Newcastle Hospitals NHS Foundation Trust (AGM, CM-B), Newcastle Upon Tyne, United Kingdom; Department of Rehabilitation and Regenerative Medicine (JM), Columbia University Irving Medical Center, New York; Neurorehabilitation Unit (VAS, EA), University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital; Unit of Neuromuscular and Neurodegenerative Disorders (ADA, ESB), Bambino Gesù Children's Hospital IRCCS, Rome; Center of Experimental and Translational Myology (CB), IRCCS Istituto Giannina Gaslini, Genoa and Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud (SSM), University of Messina, Italy; Leeds Children Hospital (A-MC); The Robert Jones and Agnes Hunt Orthopaedic Hospital (TW), Oswestry; Sheffield Children's Hospital (MTO, LS); MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre (LS), University of Oxford, United Kingdom; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liege, Belgium; Royal Hospital for Children (AM), Bristol; Royal Manchester Children's Hospital (IH); University Hospitals Birmingham (DP), United Kingdom; Evelina London Children's Hospital (VLG), London, UK; Departments of Neurology and Pediatrics (DCDV), Columbia University Irving Medical Center, New York; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School, Boston, MA; Department of Neurology (JWD), Stanford University, Palo Alto, CA; Department of Paediatrics The Children's Hospital of Philadelphia (OM, ZZ-C), Philadelphia, PA; Nemours Children's Hospital (RSF), University of Central Florida College of Medicine, Orlando; St. Jude Children's Research Hospital (RSF), Memphis, TN; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), London, United Kingdom.
Publikováno v:
Neurology. Clinical practice [Neurol Clin Pract] 2024 Jun; Vol. 14 (3), pp. e200298. Date of Electronic Publication: 2024 May 17.
Autor:
Pane M; Paediatric Neurology, Catholic University, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy., Coratti G; Paediatric Neurology, Catholic University, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy., Sansone VA; Neurorehabilitation Unit, University of Milan, Milan, Italy., Messina S; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy., Catteruccia M; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital IRCCS, Rome, Italy., Bruno C; Center of Myology and Neurodegenerative Disorders, IRCCS Istituto Giannina Gaslini, Genoa, Italy., Sframeli M; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy., Albamonte E; Neurorehabilitation Unit, University of Milan, Milan, Italy., Pedemonte M; Center of Myology and Neurodegenerative Disorders, IRCCS Istituto Giannina Gaslini, Genoa, Italy., Brolatti N; Center of Myology and Neurodegenerative Disorders, IRCCS Istituto Giannina Gaslini, Genoa, Italy., Mizzoni I; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital IRCCS, Rome, Italy., D'Amico A; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital IRCCS, Rome, Italy., Bravetti C; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy., Berti B; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy., Palermo C; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy., Leone D; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy., Salmin F; Neurorehabilitation Unit, University of Milan, Milan, Italy., De Sanctis R; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy., Pera MC; Paediatric Neurology, Catholic University, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy., Piastra M; Pediatric Intensive Care Unit, Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy., Genovese O; Pediatric Intensive Care Unit, Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy., Ricci F; AOU Città della Salute e della Scienza di Torino, Presidio OIRM (SC Neuropsichiatria Infantile), Turin, Italy., Cavallina I; AOU Città della Salute e della Scienza di Torino, Presidio OIRM (SC Neuropsichiatria Infantile), Turin, Italy., Masson R; Fondazione IRCCS Istituto Neurologico Carlo Besta Developmental Neurology Unit, Milan, Italy., Zanin R; Fondazione IRCCS Istituto Neurologico Carlo Besta Developmental Neurology Unit, Milan, Italy., Agosto C; Dipartimento di Salute della Donna e del Bambino, Università di Padova, Padua, Italy., Salomon E; Dipartimento di Salute della Donna e del Bambino, Università di Padova, Padua, Italy., Bruno I; Institute for Maternal and Child Health, IRCCS, Trieste, Italy., Magnolato A; Institute for Maternal and Child Health, IRCCS, Trieste, Italy., Bertini E; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital IRCCS, Rome, Italy., Tiziano FD; Institute of Genomic Medicine, Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy., Bovis F; Biostatistics Unit, Department of Health Sciences, University of Genoa, Genoa, Italy., Mercuri E; Paediatric Neurology, Catholic University, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy.
Publikováno v:
European journal of neurology [Eur J Neurol] 2023 Jun; Vol. 30 (6), pp. 1755-1763. Date of Electronic Publication: 2023 Apr 03.