Sarepta Reports Positive Three-Year Data for Duchenne Gene Therapy Elevidys

January 2026

Sarepta Therapeutics announced new three-year follow-up data showing that its Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec) continued to demonstrate durable functional benefits in treated patients.

According to the updated analysis, boys who received Elevidys experienced significantly slower disease progression compared with untreated natural history controls. Treated patients maintained motor function across key clinical measures, including the North Star Ambulatory Assessment (NSAA) and timed function tests, which typically decline steadily in Duchenne patients.

Photo by National Cancer Institute on Unsplash

The long-term data suggests that a single-dose gene therapy may provide sustained clinical benefit over multiple years, addressing a major unmet need in this progressive and life-limiting genetic disorder. Duchenne muscular dystrophy is characterized by ongoing muscle degeneration, often leading to loss of ambulation in early adolescence.

The announcement was positively received by investors, with Sarepta’s shares rising following the release, reflecting renewed confidence in the therapy’s long-term efficacy profile. Elevidys is currently approved in the United States for ambulatory Duchenne patients, and the new data may support broader clinical adoption and ongoing regulatory discussions.

Photo by Sangharsh Lohakare on Unsplash

Source: Reuters — Sarepta's Duchenne gene therapy slows disease progression, three-year data shows (January 26, 2026)