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Authors
Rossi, Rachele
Torelli, Silvia
Moore, Marc
Ala, Pierpaolo
Morgan, Jennifer
Malhotra, Jyoti
Muntoni, Francesco
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Funding
Funding for this research was provided by:
Sarepta Therapeutics
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Article History
Received: 12 July 2024
Accepted: 14 November 2024
First Online: 29 November 2024
Declarations
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: This work was performed under the NHS National Research Ethics: setting up of a rare diseases biological samples bank (biobank) for research to facilitate pharmacological, gene and cell therapy trials in neuromuscular disorders (REC reference number: 06/Q0406/33), and the use of cells as a model system to study pathogenesis and therapeutic strategies for Neuromuscular Disorders (REC reference 13/LO/1826).
: Not applicable.
: F. Muntoni is a member of the Rare Disease Scientific Advisory Group for Pfizer and of Dyne Therapeutics SAB and has participated in SAB meetings for PTC, Sarepta, Pfizer, Roche, Santhera, and Wave Therapeutics. UCL and Great Ormond Street Hospital have received funding from Pfizer, Italfarmaco, Wave, Santhera, Roche, NF Pharma, ReveraGen, Genethon, and Sarepta regarding clinical trials. J. Malhotra is employed by the Sarepta Therapeutics Inc.
