Document is current

Article History

Received: 29 July 2025

Accepted: 17 December 2025

First Online: 3 February 2026

Competing interests

: R.S.F. reports receiving grant support (institution) from Biogen, Dyne, Genentech, Genethon, Italfarmaco, Novartis, Roche, Sarepta and Scholar Rock; royalties from the Children’s Hospital of Philadelphia and Elsevier; advisory boards of Astellas, Biogen, Dyne, Genentech, Italfarmaco, Novartis, ReveraGen, Roche, Sarepta and Scholar Rock; honoraria and travel expenses from Novartis; patents from Roche; data safety monitoring boards of Ionis and University of Texas Southwestern; a leadership or fiduciary role for Neuromuscular Disorders (editorial board member), Journal of Neuromuscular Diseases (editorial board member) and the Pediatric Neuromuscular Clinical Research Network for SMA (steering committee member). T.O.C. reports funding from AveXis, Biogen, Catalyst, Cure SMA, Cytokinetics, Marathon, Novartis, Roche, Sarepta and the SMA Foundation; and consulting fees, advisory board and data safety monitoring board for Biogen. E.M. reports funding from AveXis, Biogen, Epirium, FamiglieSMA Italy, Ionis, Italian Telethon, NMD, Novartis, Roche and Scholar Rock. C.J.S. reports grant support from Actio Biosciences, Biogen and Roche; royalties from Elsevier; honoraria from Biogen, Genentech, Regeneron and Roche; travel to meetings support from Biogen, Regeneron and Roche; data safety monitoring board for Biogen; leadership or fiduciary role for the Peripheral Nerve Society (President); advisory boards for Cure SMA, the Charcot–Marie–Tooth Association, the Charcot–Marie–Tooth Research Foundation, the Kennedy’s Disease Association, the Muscular Dystrophy Association and the Packard Center and Merkin Center; and receipt of drugs from Ionis and Roche. M.d.M.G.R. reports honoraria, expert testimony, travel expenses and data safety monitoring board support from Biogen. J.W.D. reports grant support (institution) from AMO Pharma, AnnJi Pharmaceutical, Astellas Gene Therapies, Avidity Biosciences, Biogen, CureSMA, Genentech, Ionis, the Muscular Dystrophy Association, the Myotonic Dystrophy Foundation, NMD Pharma, Novartis, Roche, Sanofi, Sarepta, Scholar Rock and the SMA Foundation; royalties (individual and institution) from Athena Diagnostics; and consulting fees from Avidity Biosciences, Biogen, Epirium Bio, Kate Therapeutics, Novartis, PepGen, Roche/Genentech Pharmaceuticals, Sanofi, Sarepta Therapeutics, Scholar Rock, Solid Biosciences and Vertex Pharmaceuticals. J.M. reports grant support (institution) from Genentech and Scholar Rock; honoraria from Roche and Scholar Rock; and data safety monitoring boards for Argenx, Biogen, Roche and Scholar Rock. P.S., B.T., A.D.P., E.B., J.I., R.L., G.G., R. Foster, R. Farewell and S.F. are employees of Biogen and may hold stock. J.S. and M.M. were employees of Biogen at the time of contributing to this work.