Sarepta Therapeutics, Inc. has announced positive topline three-year functional results from Part 1-treated patients in EMBARK, the company’s global, randomized placebo-controlled Phase 3 study evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory individuals living with Duchenne. According to Sarepta, participants were aged four to seven at time of treatment and were on average over nine years old at time of last assessment.

Results indicate that patients who received ELEVIDYS in Part 1 of EMBARK demonstrated statistically significant efficacy across key motor function measures–North Star Ambulatory Assessment (NSAA), Time to Rise (TTR) and 10-meter walk/run (10MWR)–when compared to an untreated external control (EC) group*. Further, Sarepta highlights that patients treated with ELEVIDYS in Part 1 showed an increasing treatment effect over time and maintained significantly higher levels of motor function three years after treatment compared to the EC group.

Data shows the mean NSAA score remained above baseline at Year 3 for the ELEVIDYS-treated group (n=52) while the EC group (n=73) continued to show the expected age-related decline below baseline. Additionally, the ELEVIDYS group showed a 73% slowing of disease progression as measured by TTR and 70% slowing of disease progression as measured by 10MWR when compared to the EC group.

According to Sarepta, no new treatment-related safety signals were observed. The company reports that ongoing analysis of the three-year data includes functional results from crossover-treated patients two years after treatment. PPMD will share future updates with the community as information becomes available.

Read Sarepta’s press release here and community letter here.

*The pre-specified external control analysis included data from three separate studies in Duchenne, comprising DMD controls from one randomized trial and two natural history cohorts who met predefined matching criteria. Comparison of treated and control patients was based on a pre-specified, propensity score weighting approach using age, height, BMI, steroid usage, baseline NSAA and timed function tests in order to balance key prognostic factors between the groups.

BREAKING NEWS WEBINAR

Sarepta joined PPMD for a breaking news community webinar on January 26, 2026, to discuss these topline results and answer questions from the community. Watch the recording: