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Article History

Received: 22 August 2025

Accepted: 6 October 2025

First Online: 3 November 2025

Declarations

:

: Ivana Audhya, Alise B. Nacson, and Katherine Gooch are employees of Sarepta Therapeutics, Inc., and may hold shares and/or stock options in the company. Bonita Basnyat, Christina Slota, and Susan Martinare full-time employees of RTI-HS, a subsidiary of RTI International, an independent nonprofit research organization, which was retained by Sarepta Therapeutics, Inc., to conduct the research that is the subject of this manuscript; their compensation is unconnected to the studies on which they work. Alex Murphy, Claire J. Lansdall, and Teofil Ciobanu are full-time employees and minor shareholders of F. Hoffmann-La Roche AG. Andres Nascimento has received fees from AveXis, Biogen, and F. Hoffmann-La Roche, Ltd. Aravindhan Veerapandiyan has a consultancy/advisory role with AMO Pharma, AveXis, Biogen, Edgewise Therapeutics, FibroGen, Novartis, Pfizer, PTC Therapeutics, Sarepta Therapeutics, UCB Pharma, Catalyst, and Scholar Rock; has received research funding from AMO Pharma, Capricor Therapeutics, Edgewise Therapeutics, FibroGen, the Muscular Dystrophy Association, Novartis, Parent Project Muscular Dystrophy, Pfizer, RegenxBio, and Sarepta Therapeutics; and has other relationship(s) with MedLink Neurology for editorial services.

: The main EMBARK trial protocol, including the optional, qualitative in-trial interview substudy, was approved by an institutional review board and ethics committee at each site. The full list of institutional review boards and ethics committees has been published [ ], and the trial was conducted in accordance with both the Declaration of Helsinki and Good Clinical Practice guidelines [ ]. All eligible caregivers provided informed consent to participate in EMBARK [ ] and additional consent to participate in the interview substudy and be audio-recorded. Interview participants were aware that their responses may be collated and published.