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Authors
McDonald, Craig M.
Elkins, Jacob S.
Dharmarajan, Sai
Gooch, Katherine
Ciobanu, Teofil
Lansdall, Claire J.
Murphy, Alexander P.
McDougall, Fiona
Mercuri, Eugenio M.
Audhya, Ivana
,
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Clinical Trials BETA
Clinical trials referenced in this document:
nct05096221 at ClinicalTrials.govDocuments that mention this clinical trial
Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy
https://doi.org/10.1007/s40120-024-00685-8
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Funding
Funding for this research was provided by:
Sarepta Therapeutics
F. Hoffmann-La Roche
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Article History
Received: 23 August 2024
Accepted: 7 November 2024
First Online: 26 November 2024
Declarations
:
: Jacob S. Elkins, Sai Dharmarajan, Katherine Gooch, Ivana Audhya: Employees of Sarepta Therapeutics, Inc., and may hold stock/options in the company. Teofil Ciobanu: Employee of F. Hoffmann-La Roche Ltd. Claire J. Lansdall: Employee of F. Hoffmann-La Roche Ltd. and shareholder of F. Hoffmann-La Roche Ltd. Alexander P. Murphy: Employee of Roche Products UK and may hold shares in F. Hoffmann-La Roche Ltd. Fiona McDougall: Employee of Genentech, Inc.and shareholder of F. Hoffmann-La Roche Ltd. Craig M. McDonald: Reports grants from Capricor Therapeutics, Catabasis, Edgewise Therapeutics, Epirium Bio, Italfarmaco, Pfizer, PTC Therapeutics, Santhera Pharmaceuticals, Sarepta Therapeutics; and has a consultancy/advisory role with Biomarin, Capricor Therapeutics, Catalyst, Edgewise Therapeutics, Italfarmaco, PTC Therapeutics, F. Hoffmann-La Roche Ltd, Santhera Pharmaceuticals and Sarepta Therapeutics. He has received honoraria from PTC Therapeutics and Sarepta Therapeutics. Eugenio M. Mercuri: Reports receiving fees from AveXis, Biogen, and F. Hoffmann-La Roche Ltd.
: EMBARK was conducted in accordance with the provisions of the Declaration of Helsinki and Good Clinical Practice guidelines. Trial protocol and all amendments were approved by an institutional review board and ethics committee at each site. The full list of institutional review boards and ethics committees is available in the Supplementary Information. Informed consent, including consent to publish, was obtained from parent(s)/legal guardian(s), and patients’ assent was obtained when indicated.
