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Authors
Butterfield, Russell J. https://orcid.org/0000-0002-8452-7189
Shieh, Perry B. https://orcid.org/0000-0001-7145-7663
Li, Huihua
Binks, Michael
McDonnell, Tara G.
Ryan, Kelly A.
Delnomdedieu, Marielle
Belluscio, Beth A.
Neelakantan, Srividya
Levy, Daniel I. https://orcid.org/0000-0003-0603-0297
Schwartz, Pamela F.
Smith, Edward C.
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Funding
Funding for this research was provided by:
Pfizer
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Article History
Received: 22 June 2023
Accepted: 1 May 2025
First Online: 27 June 2025
Competing interests
: R.J.B. has received support from or has served as a consultant for Sarepta Pharmaceuticals, Scholar Rock, AveXis, Pfizer, Biogen, Reata and Aavanti. P.B.S. has received contracted research support from Pfizer; his institution has received contracted research funding from Sarepta, Solid Biosciences, ReveraGen, PTC Therapeutics, Astellas Gene Therapies, Novartis Gene Therapies, Santhera, Biogen, AMO Pharma and Sanofi. He has served on advisory boards for Sarepta, Alexion, Argenx, Biogen and Genentech, and has received speaker fees from Alexion, Catalyst, Grifols, Genentech, CSL Behring, Biogen and Argenx. H.L., K.A.R., M.D., S.N., D.I.L. and P.F.S. are employees of Pfizer and hold stock or stock options. M.B. and T.G.M. were employees of Pfizer and held stock or stock options at the time of the study. M.B. and T.G.M. are also named on a patent (no. WO2020261178A1) applied for ‘Methods of treating Duchenne muscular dystrophy using AAV mini-dystrophin gene therapy’ but receive no direct financial benefit. B.A.B. (currently retired) was an employee of Pfizer at the time of the study. E.C.S. has received partial salary support from Pfizer for his role as the principal investigator of the study and has served as a paid consultant for Pfizer, Sarepta, NSPharma, Lilly, REGENXBIO, Entrada, Solid Biosciences and Edgewise Therapeutics.
