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The authors declare the following competing interests. C.M.P. has served as a consultant for Novartis Gene Therapies, Inc.\/Novartis Pharma AG, Biogen and Sarepta; has served on a speaker\u2019s bureau for Novartis Gene Therapies, Inc.\/Novartis Pharma AG and Biogen; and has received research support from Novartis Gene Therapies, Inc.\/Novartis Pharma AG, Biogen, Sarepta, PTC, CSL Behring, Scholar Rock and Catabasis. J.M.W. has received fees from Roche; serves on the national South African advisory boards for Novartis and Sanofi; and is chief editor for the Pediatric Neurology section of\n                      Frontiers in Neurology\n                      . O.S. has received research grants from Novartis and Thermo Fisher Scientific, received honoraria for lectures and speaker\u2019s bureau from F. Hoffmann-La Roche and Novartis and serves as a board member of the Foundation to Eradicate Neuromuscular Diseases (FEND) and on an advisory panel for the Thai SMA group. S.G., H.X., H.C.M., S.K.H.T., M.-K.T., A.P.B., A.B.O., M.A.A.-M. and D.C.V. have no conflicts related to this work to disclose. Y.-J.J. has participated in clinical trials with Biogen, Novartis, Roche, PTC, Sarepta and Pfizer; received speaker and\/or consulting fees from Biogen, Novartis, Roche and Pfizer; and received research grants from Biogen. A.W.L., J.V., S.T.-W., I.A. and R.P. are employees of Novartis and own stock\/other equities. R.S.F. has received personal compensation for advisory board\/data safety monitoring board participation from Astellas, Dyne, Italfarmaco, AveXis\/Novartis Gene Therapies, Inc.\/Novartis Pharma AG, NS Pharma, Biogen, Catabasis, Ionis, Italfarmaco, ReveraGen, Roche\/Genentech, Sarepta, Satellos and Scholar Rock; editorial fees from Elsevier for co-editing a neurology textbook; license fees from the Children\u2019s Hospital of Philadelphia; and research support from Biogen and Roche\/Genentech. R.S.F. also participated as an investigator in clinical trials sponsored by Novartis Gene Therapies, Inc.\/Novartis Pharma AG, Biogen, Catabasis, Dyne, Genethon, Italfarmaco, ReveraGen, Roche\/Genentech, Sarepta and Scholar Rock.","order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}]}}