Sarepta to Share Gene Therapy Data at 2026 Neuromuscular Meeting

Sarepta Therapeutics Inc. will present data from its gene therapy pipeline at the 27th Annual Neuromuscular Study Group Scientific Meeting in San Antonio, Texas, from September 25–27, 2026. The company will highlight results from the Phase 4 ENDURE study, which examines delandistrogene moxeparvovec (marketed as ELEVIDYS) in real-world clinical settings for Duchenne muscular dystrophy patients receiving prophylactic immunosuppression with sirolimus. Five posters will focus on clinical validation of micro-dystrophin expression, treatment delays, real-world safety profiles, mobility improvements, and postmarketing surveillance for ELEVIDYS, a single-dose gene therapy approved for ambulatory patients aged four and older with confirmed DMD gene mutations. Notably, ELEVIDYS carries a boxed warning for acute liver injury and contraindications for patients with deletions in exons 8 and 9 of the DMD gene, as well as restrictions for those with preexisting liver impairment, recent vaccinations, or active infections. Common adverse reactions include vomiting, nausea, liver injury, fever, thrombocytopenia, and elevated troponin-I levels. Additionally, Sarepta will report interim safety and exposure data from Phase 1/2 trials of SRP-1003 for myotonic dystrophy type 1 and SRP-1001 for FSHD1, targeting the αvβ6 integrin pathway.