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EXONDYS 51 (eteplirsen)

Duchenne Muscular Dystrophy (DMD) - exon 51 amenable

ApprovedCommercial

Key Facts

Indication
Duchenne Muscular Dystrophy (DMD) - exon 51 amenable
Phase
Approved
Status
Commercial
Company

About Sarepta Therapeutics

Sarepta Therapeutics is a pioneering, commercial-stage biotech focused on developing and commercializing precision genetic medicines for devastating rare diseases, primarily Duchenne muscular dystrophy. The company has multiple FDA-approved exon-skipping therapies on the market and is advancing a robust pipeline that includes a commercial gene therapy and other candidates for DMD and limb-girdle muscular dystrophy (LGMD). With a hybrid manufacturing strategy and a relentless focus on following the science, Sarepta aims to reclaim futures for patients impacted by rare genetic disorders.

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