SRP-9003
Limb-Girdle Muscular Dystrophy Type 2E/R4
Phase 3Active (Gene Therapy)
Key Facts
Indication
Limb-Girdle Muscular Dystrophy Type 2E/R4
Phase
Phase 3
Status
Active (Gene Therapy)
Company
About Sarepta Therapeutics
Sarepta Therapeutics is a commercial-stage biotech leader with an urgent mission to engineer and deliver precision genetic medicines for rare diseases. The company has achieved significant commercial success with multiple FDA-approved RNA-based exon-skipping therapies for Duchenne muscular dystrophy (DMD) and the landmark approval of ELEVIDYS, a gene therapy for DMD. Its strategy leverages a deep, multi-modal pipeline, a hybrid manufacturing model for scale, and a global commercial footprint to solidify its position as a dominant force in rare disease therapeutics.
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