Measuring Broad and Meaningful Impacts With Patient‐Reported Outcomes in Spinal Muscular Atrophy as a Framework for Neuromuscular Disorders

The authors have nothing to report. M.F. is a site principal investigator for Roche and Novartis Gene Therapies Inc. SMA clinical trials, and the institution has received funds for contract research related to the conduct of these trials. M.F. has received honoraria for advisory board participation and participation in educational events from Biogen, Novartis, and Roche, speaker's fees from Biogen and Novartis and serves as a medical director on the board of Muscular Dystrophy NSW (not-for-profit). The authors have nothing to report.