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Authors
Duong, Tina https://orcid.org/0000-0003-3177-4722
Haselkorn, Tmirah
Miller, Beckley
Coats, Julie
Jensen, Ivar
Ward, Erin
Wood, Marie
Graham, Robert J.
Servais, Laurent
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Funding
Funding for this research was provided by:
Astellas Gene Therapies
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Article History
Received: 17 April 2024
Accepted: 28 January 2025
First Online: 12 May 2025
Declarations
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: The survey design and content were reviewed and approved by the Advarra Institutional Review Board to be used for scientific purposes. The survey was conducted according to the principles expressed in the Declaration of Helsinki.
: Written informed consent was obtained from all participants.
: TD is a consultant for Astellas Gene Therapies, Dyne Therapeutics, Avidity, Roche; and has participated in advisory boards for Roche, Novartis, and Biogen. TH and JC are employees of Astellas Gene Therapies. BM and IJ are employees of PRECISIONheor, a division of Precision Medicine Group, which received funding from Astellas Gene Therapies (formerly Audentes Therapeutics, Inc.) for this research. EW and MW report no competing interests. RJG reports being on the advisory board of Astellas Pharmaceuticals for X-linked myotubular myopathy and Pompe disease outside the submitted work. LS has received consulting fees from Sarepta, Pfizer, Roche, Affina, Renegex Bio, Astellas Gene Therapies (formerly Audentes Therapeutics, Inc.), Evox Therapeutics, and Novartis; has received honoraria from Sarepta, Roche, Audentes Therapeutics, Inc., and Novartis; and is the Secretary of the World Muscle Society.
