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BioWorld - Sunday, May 18, 2025
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Home » Adcom briefing docs mixed; will FDA see Sarepta’s ‘grander vision’ in DMD gene therapy?
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Adcom briefing docs mixed; will FDA see Sarepta’s ‘grander vision’ in DMD gene therapy?

May 10, 2023
By Randy Osborne
How grave they might be remains unknown, but regulatory questions have surfaced in briefing documents related to the soon-to-happen panel meeting on Sarepta Therapeutics Inc.’s gene transfer therapy delandistrogene moxeparvovec in Duchenne muscular dystrophy (DMD). The U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee will meet May 12 to discuss the compound, also known as SRP-9001.
BioWorld Regulatory Musculoskeletal Gene therapy FDA

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