Cambridge, Mass. — Sarepta Therapeutics will present new clinical and real-world data from its neuromuscular disease portfolio at the 27th Annual Neuromuscular Study Group Scientific Meeting, taking place Sept. 25-27 in San Antonio, Texas.
The company plans to present seven posters, including the first results from the Phase 4 ENDURE study evaluating delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy who received the treatment as part of routine clinical care.
The ENDURE analysis includes safety data from patients who received prophylactic immunosuppression with sirolimus.
“The data being presented at NMSG 2026 provide new insights into the real-world use of delandistrogene moxeparvovec and add to the growing body of evidence supporting its clinical profile,” said Louise Rodino-Klapac, Ph.D., President, Research & Development and Technical Operations at Sarepta. “Observations from clinical practice are especially valuable because they help us better understand how treatment performs across a broader range of patients and care settings, and we are encouraged that these findings continue to reinforce our understanding of both the safety and effectiveness of delandistrogene moxeparvovec.”
Sarepta will also present analyses examining the relationship between micro-dystrophin expression, muscle MRI findings and functional outcomes, as well as the potential impact of treatment delays in ambulatory Duchenne patients.
Other presentations will include postmarketing safety data for delandistrogene moxeparvovec and mobility outcomes from Part 2 of the EMBARK study.
The company will also present interim Phase 1/2 findings for two investigational siRNA therapies. SRP-1003 is being evaluated in patients with myotonic dystrophy type 1, while SRP-1001 is being studied in patients with facioscapulohumeral muscular dystrophy type 1.
All seven posters are scheduled to be presented during the NMSG poster session on Sept. 25.
Sarepta’s ELEVIDYS, or delandistrogene moxeparvovec-rokl, is a single-dose AAV-based gene transfer therapy designed to address the underlying genetic cause of Duchenne muscular dystrophy by delivering a transgene that enables production of micro-dystrophin in skeletal muscle.
ELEVIDYS is indicated for ambulatory patients ages 4 and older with Duchenne muscular dystrophy who have a confirmed mutation in the DMD gene.
