Conflicts of interest
CM, AveXis/Novartis
MO reports grants from F. Hoffmann-La Roche Ltd during the conduct of the study and has received grants as a clinical trial investigator from Biogen. JWD has received fees for serving on scientific advisory boards from Biogen, Novartis, Sarepta Therapeutics, and Avidity; has received consulting fees from Affinia Therapeutics and Shift Therapeutics for a therapeutic platform; and has received grant support for clinical trials from F. Hoffmann-La Roche Ltd, Biogen, Novartis Gene Therapies, Cytokinetics, Scholar Rock, and Sarepta Therapeutics. ND served on scientific advisory boards for F. Hoffmann-La Roche Ltd, Biogen and Novartis Pharmaceuticals; he has received personal fees from Biogen and F. Hoffmann-La Roche Ltd for congress and travel support. ESM reports that she has served on advisory boards for Biogen and Scholar Rock. She has received consulting fees, travel support, and speaker honoraria as an independent contractor from F. Hoffmann-La Roche Ltd, Biogen, AveXis, and Scholar Rock. AN has received fees for serving on scientific advisory boards and speaker fees from F. Hoffmann-La Roche Ltd. KS reports grants from F. Hoffmann-La Roche Ltd/Chugai Pharmaceutical during the conduct of the study and grants from Biogen Japan and lecture fees from Novartis Pharmaceuticals, outside the submitted work. CV reports personal fees and financial support to her institution from F. Hoffmann-La Roche Ltd for activities outside the submitted work. GB received consultancy fees and speaker honoraria from F. Hoffmann-La Roche Ltd, AveXis/Novartis Gene Therapy, and Biogen, and grants from F. Hoffmann-La Roche Ltd. NG reports fees for serving on advisory boards and presentations at symposia from F. Hoffmann-La Roche Ltd, Biogen, AveXis, and Novartis. JK received grants or contracts from Novartis Gene Therapies and Biogen. He has received consulting and speaker fees from F. Hoffmann La Roche Ltd, Biogen, and Novartis Gene Therapies and consulting fees from Scholar Rock. AK-P reports that she received an institutional support grant from Biogen; serving on scientific advisory boards for and receiving speaker honoraria from Biogen, F. Hoffmann-La Roche Ltd, Novartis, and PTC Therapeutics; and receiving personal fees from Biogen and F. Hoffmann-La Roche Ltd for travel support. LS received grants and personal fees from F. Hoffmann-La Roche Ltd, Biogen, and AveXis/Novartis Gene Therapies and personal fees from Cytokinetics, BioHaven, and Scholar Rock, outside the submitted work. GP, KG, HK, CM, TM, RSS, HS, WYY, and PF report that they are current employees of and stockholders in F. Hoffmann-La Roche Ltd. EM has received fees for serving on scientific advisory boards; speaker fees from F. Hoffmann-La Roche Ltd, Biogen, AveXis/Novartis, Scholar Rock, and Cytokinetics; and grants from Biogen during the conduct of the study.