Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on genetic medicines for rare neuromuscular diseases, primarily Duchenne muscular dystrophy (DMD), a fatal X-linked disease that causes progressive muscle deterioration in boys due to the absence of the protein dystrophin. Sarepta sells four approved DMD products in the U.S. The first three — EXONDYS 51, VYONDYS 53, and AMONDYS 45 — are weekly-infused exon-skipping therapies that partially restore a truncated but functional form of dystrophin; each targets a different patient subset based on genetic mutation. The fourth, ELEVIDYS, is a one-time AAV-based gene therapy that delivers a shortened dystrophin gene in a single infusion, approved for ambulatory patients aged four and older. Outside the U.S., Roche commercializes ELEVIDYS in exchange for royalties and milestones paid to Sarepta. The PMO franchise generates stable, recurring revenue since patients remain on therapy indefinitely, while ELEVIDYS revenue is driven entirely by new patient starts each quarter, creating significant variability. The ELEVIDYS patient journey — involving insurance authorization, antibody testing, and specialized infusion — takes four to six months and resembles an organ transplant more than a typical prescription. Sarepta relies on contract manufacturers for production and has no internal GMP manufacturing. Looking ahead, Sarepta's primary growth bet is a broad siRNA pipeline developed in collaboration with Arrowhead Pharmaceuticals, targeting diseases including myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy (FSHD), with early proof-of-concept data expected in early 2026.