CAMBRIDGE, Mass. — September 24, 2026

Sarepta Therapeutics, Inc. announced that it will present seven posters covering its neuromuscular medicine portfolio at the 27th Annual Neuromuscular Study Group Scientific Meeting in San Antonio, Texas, from September 25-27, 2026. The presentations include the first results from the Phase 4 ENDURE study, a prospective, multicenter observational study evaluating real-world use of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD), including safety findings in patients receiving prophylactic immunosuppression with sirolimus.

Sarepta Presents Real-World ELEVIDYS Data in Duchenne

Five presentations will focus on delandistrogene moxeparvovec, marketed as ELEVIDYS, in DMD. The Phase 4 ENDURE interim analysis evaluates safety following real-world treatment, while additional analyses examine the relationship between micro-dystrophin expression, muscle MRI and functional outcomes; treatment delays in ambulatory patients; PROMIS parent-proxy mobility scores compared with external controls; and the U.S. postmarketing safety profile. These analyses are intended to expand the clinical evidence base for ELEVIDYS beyond controlled clinical development and provide information on treatment use across broader clinical-care settings.

Portfolio Expands Into DM1 and FSHD

Sarepta will also present interim findings from two Phase 1/2 programs targeting myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy type 1 (FSHD1). SRP-1003 is being evaluated as an αvβ6 integrin-targeting siRNA therapy for DM1, while SRP-1001 is an αvβ6 integrin-targeting siRNA therapy being studied in FSHD1. The presentations will report safety and exposure findings from these early-stage clinical programs, adding to Sarepta’s efforts to develop genetic medicines across multiple neuromuscular diseases.

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ELEVIDYS Remains a Single-Dose Gene Transfer Therapy

ELEVIDYS is an AAV-based gene transfer therapy administered as a single intravenous infusion and is designed to deliver a transgene that supports production of micro-dystrophin in skeletal muscle. The therapy is FDA-approved for ambulatory patients aged 4 years and older with DMD and a confirmed DMD gene mutation. Its prescribing information includes a boxed warning for acute serious liver injury and acute liver failure, along with warnings concerning serious infections, myocarditis, infusion-related reactions, immune-mediated myositis and preexisting immunity to AAVrh74. The NMSG presentations will provide additional clinical and real-world information as Sarepta continues to evaluate ELEVIDYS and advance its broader neuromuscular pipeline.

Source :Sarepta Therapeutics, press release