Sherlock, Sarah P.
Palmer, Jeffrey
Wagner, Kathryn R.
Abdel-Hamid, Hoda Z.
Tian, Cuixia
Mah, Jean K.
Muntoni, Francesco
Guglieri, Michela
Butterfield, Russell J.
Charnas, Lawrence
Marraffino, Shannon
Article History
Received: 11 March 2022
Accepted: 25 October 2022
First Online: 5 November 2022
Competing interests
: SPS, JP, LC, and SM are employees of and hold stock or stock options in Pfizer. KRW is an employee of and holds stock in F. Hoffmann-La Roche Ltd. HZA-H has received research support from and served on advisory boards for Sarepta Therapeutics, Biogen, NS Pharma, and AveXis/Novartis. CT has been site investigator for clinical trials with Pfizer, AveXis/Novartis, Catabasis, WaVe, Roche, PTC Therapeutics, FibroGen, Capricor, Santhera, Sarepta Therapeutics, and Summit. JKM has received research support for clinical trials from Pfizer, Italfarmaco, Sarepta Therapeutics, Catabasis, Roche, Biogen, Novartis, NS Pharma, PTC Therapeutics, and ReveraGen; and consultant fees from PTC Therapeutics, Roche, and Biogen. FM is a member of the Pfizer Rare Disease Scientific Advisory Board; received grant support from Biogen and Sarepta Therapeutics; and received consultant fees and/or speakers’ honoraria from Novartis, Biogen, Roche, Dyne Therapeutics, and PTC Therapeutics. MG has served as chair for a ReveraGen study; collaborated on research with ReveraGen and Sarepta Therapeutics; acted as principal investigator for clinical trials sponsored by Pfizer, Italfarmaco, Santhera Therapeutics, ReveraGen, Dynacure, Roche, PTC Therapeutics, and Summit; participated in advisory boards for Pfizer, NS Pharma, and Dyne (consultancies through Newcastle University); and performed consultancy work (speaker) for Sarepta Therapeutics. RJB has received funding via contracts for clinical trials from AveXis/Novartis, PTC Therapeutics, Sarepta Therapeutics, Pfizer, and Biogen; and has served on scientific advisory boards for Sarepta Therapeutics, Biogen, AveXis/Novartis, and Pfizer.