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Article History

Received: 23 July 2025

Accepted: 4 November 2025

First Online: 8 December 2025

Competing interests

: Novartis Pharma AG sponsored this clinical trial. 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’s 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 Frontiers in Neurology . O.S. has received research grants from Novartis and Thermo Fisher Scientific, received honoraria for lectures and speaker’s 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’s 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.