Sarepta Therapeutics has reported three-year data showing slowed disease progression for its gene therapy Elevidys in patients with Duchenne muscular dystrophy (DMD), reinforcing the long-term clinical and commercial potential of the treatment.

The results from the Phase 3 EMBARK study showed that ambulatory patients treated with Elevidys maintained better motor function compared with an external control group over three years. Key mobility measures indicated durable benefit following the one-time gene therapy administration.

Elevidys, also known as delandistrogene moxeparvovec, is designed to address the genetic cause of DMD by enabling muscle cells to produce a shortened dystrophin protein. The therapy is currently approved for ambulatory patients aged four years and older.

Sarepta reported that no new safety signals were observed during the three-year follow-up, supporting the therapy’s existing safety profile. More than 1,200 patients have been treated globally to date.

The long-term data strengthen Elevidys’ role in Sarepta’s rare disease and gene therapy portfolio, where durable outcomes are critical for regulatory confidence, payer acceptance and sustained adoption.

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