Sarepta Therapeutics Inc (NASDAQ: SRPT), a provider of precision genetic medicine for rare diseases, on Monday announced positive topline three-year results from Part 1 of its global Phase 3 EMBARK trial evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory patients with Duchenne muscular dystrophy (DMD). At a mean age of nine, patients treated with ELEVIDYS (n=52) maintained North Star Ambulatory Assessment (NSAA) scores above baseline, demonstrating durable functional benefit three years after treatment.

ELEVIDYS-treated patients showed a 70% or greater reduction in disease progression compared with a propensity-weighted external control group, as measured by Time to Rise (TTR) and 10-metre walk/run (10MWR). The functional gap versus controls widened between Years 2 and 3, indicating an increasing treatment effect over time. Topline data showed: NSAA improvement of +4.39 points (p=0.0002), TTR improvement of -6.05 seconds (p