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# Sarepta Reports Positive Data on Duchenne Gene Therapy
- URL: https://www.fdaweb.com/sarepta-reports-positive-data-on-duchenne-gene-therapy/
- Published: 2026-01-26T12:00:00.000Z
- Updated: 2026-09-14T15:31:32.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5160542

Sarepta Therapeutics is announcing that its gene therapy Elevidys significantly slowed disease progression over three years in ambulatory patients with Duchenne muscular dystrophy, according to topline results from the Phase 3 EMBARK trial. The company says patients treated with Elevidys maintained higher levels of motor function than untreated controls across multiple measures, including the North Star Ambulatory Assessment, time to rise, and the 10-meter walk/run. Sarepta described the results as statistically significant, clinically meaningful, and durable.

According to Sarepta, the mean North Star Ambulatory Assessment score in the treated group remained above baseline three years after dosing, while scores in a pre-specified, propensity-weighted external control group continued to decline with age. The company reported a 73% slowing in disease progression as measured by time to rise and a 70% slowing as measured by the 10-meter walk/run compared with the external control group.

Sarepta said the treatment effect increased over time, with a widening functional gap between Elevidys-treated patients and controls between years two and three. No new treatment-related safety signals were observed, the company said, and the three-year safety profile was consistent with prior experience in ambulatory patients.

“Elevidys is the first gene therapy for Duchenne to show a dramatic shift in disease trajectory out to three years in a robust, controlled clinical dataset,” Sarepta’s president of research and development **Louise Rodino-Klapac** said in a statement.

The company plans to present the data at upcoming medical meetings and in peer-reviewed publications. Two-year EMBARK results were published earlier this month in *Neurology & Therapy*.

Elevidys is currently the only approved gene therapy for Duchenne muscular dystrophy. The therapy has been administered to more than 1,200 patients worldwide in clinical trials and real-world settings, according to Sarepta. In late 2025, FDA updated the U.S. label to allow use in ambulatory patients aged four and older.