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FeaturedRare Disease

Delandistrogene moxeparvovec (Elevidys)

Sarepta Therapeutics

Gene therapy delivering micro-dystrophin. FDA approved under accelerated approval. EMBARK confirmatory trial ongoing.

Duchenne Muscular Dystrophy

Linked Studies

NCT03375255PrimaryCOMPLETEDPHASE1

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)

Muscular Dystrophy, DuchenneUpdated Jul 2022
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Also Known As

Other names

delandistrogene
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