Sarepta reports ELEVIDYS Duchenne trial data

• Sarepta Therapeutics (NASDAQ:SRPT) presented new ELEVIDYS data showing functional benefits in older ambulatory patients with Duchenne muscular dystrophy.
• Patients aged 8–12 treated with ELEVIDYS showed sustained functional outcomes over two years compared with a matched external control group.
• Sarepta reported additional early-age data showing dystrophin expression after treatment in patients aged 2–3 years.

Sarepta Therapeutics (NASDAQ:SRPT) presented new clinical data showing ELEVIDYS (delandistrogene moxeparvovec) was associated with functional benefits in older ambulatory patients with Duchenne muscular dystrophy at the World Muscle Society Congress 2026.

The analysis included 25 patients aged 8–12 years from the EMBARK and ENDEAVOR studies compared with 99 matched external control patients, with treated patients showing differences across functional measures over two years.

"The period between 8 and 12 years of age represents a critical phase of Duchenne when many boys transition from relative stability to progressive functional decline," said Sarepta President of Research & Development and Technical Operations Louise Rodino-Klapac, Ph.D.

The study reported clinically meaningful results across North Star Ambulatory Assessment (NSAA) total scores, rise-from-floor velocity and 10-meter walk/run velocity at one and two years after infusion, while safety findings remained consistent with the known safety profile of ELEVIDYS.

Sarepta also presented an analysis from the ENDEAVOR and ENVOL studies showing robust delandistrogene moxeparvovec-dystrophin expression and sarcolemmal localization 12 weeks after infusion in ambulatory patients aged 2–3 years.

ELEVIDYS is a gene therapy developed for Duchenne muscular dystrophy, and Sarepta stated that continued patient follow-up will be needed to understand longer-term functional outcomes across different treatment ages.