Sherlock, Sarah P. http://orcid.org/0000-0001-5351-3828
Palmer, Jeffrey
Wagner, Kathryn R.
Abdel-Hamid, Hoda Z.
Bertini, Enrico
Tian, Cuixia
Mah, Jean K.
Kostera-Pruszczyk, Anna
Muntoni, Francesco
Guglieri, Michela
Brandsema, John F.
Mercuri, Eugenio
Butterfield, Russell J.
McDonald, Craig M.
Charnas, Lawrence
Marraffino, Shannon
Funding for this research was provided by:
Pfizer
Article History
Received: 23 November 2021
Revised: 11 March 2022
Accepted: 12 March 2022
First Online: 8 April 2022
Declarations
:
: 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. HZAH has received research support from and served on advisory boards for Sarepta Therapeutics, Biogen, NS Pharma, and AveXis/Novartis. EB has served on scientific advisory boards for Sarepta Therapeutics, Biogen, AveXis, and PTC Therapeutics. CT has been site investigator for clinical trials with Pfizer, AveXis/Novartis, Catabasis, WaVe, Roche, PTC Therapeutics, Fibrogen, Capricor, Santhera Therapeutics, 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 Roche and Biogen. AKP has served on advisory boards for or received consultant fees from Pfizer, Biogen, AveXis, Novartis, PTC Therapeutics, and Roche; and received research support from Biogen, Sanofi-Aventis, CSL Behring, Kedrion, and Shire. FM is a member of the Pfizer Rare Disease Scientific Advisory Board; has received grant support from Biogen and Sarepta Therapeutics; and has 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 preformed consultancy work (speaker) for Sarepta Therapeutics. JFB has been site investigator for clinical trials with Alexion, AveXis/Novartis, Biogen, Catabasis, CSL, Behring, Cytokinetics, Fibrogen, Ionis, Pfizer, PTC Therapeutics, Sarepta Therapeutics, Summit, and WaVe; consultant for Audentes, AveXis/Novartis, Biogen, Cytokinetics, Genentech, Marathon, Momenta, NS Pharma, PTC Therapeutics, Sarepta Therapeutics, Scholar Rock, and WaVe; speaker for AveXis/Novartis and Biogen; and medical advisory council member for Cure SMA. EM has served on advisory boards for Sarepta Therapeutics, Santhera Therapeutics, PTC Therapeutics, Pfizer, NS Pharma, and Roche. RJB has received funding via contracts for clinical trials from AveXis/Novartis, PTC Therapeutics, Sarepta Therapeutics, Pfizer, and Biogen; and served on scientific advisory boards for Sarepta Therapeutics, Biogen, AveXis/Novartis, and Pfizer. CMM has acted as a consultant on clinical trials for Astellas, Avidity Biosciences, BioMarin, Capricor Therapeutics, Catabasis Pharmaceuticals, Edgewise Therapeutics, Eli Lilly and Company, Entrada Therapeutics, Epirium Bio (formerly Cardero Therapeutics), FibroGen, Gilead, Hoffmann-La Roche, Italfarmaco, Pfizer, PTC Therapeutics, Santhera Pharmaceuticals, and Sarepta Therapeutics; and received research support for clinical trials from BioMarin, Capricor Therapeutics, Eli Lilly and Company, Italfarmaco, Pfizer, PTC Therapeutics, Hoffmann-La Roche, Santhera Pharmaceuticals, and Sarepta Therapeutics.
: The original phase 2 trial was conducted in accordance with legal and regulatory requirements, as well as the general principles set forth in the International Ethical Guidelines for Biomedical Research Involving Human Subjects, guidelines for Good Clinical Practice, and the Declaration of Helsinki. The protocols, any amendments, and informed consent/assent documents were approved by the institutional review board or ethics committee at each study center.
: A parent or legal guardian provided written informed consent prior to any study-specific activity that was performed.
: Not applicable.