Sarepta Therapeutics

Stories

The race to build a better Duchenne therapy

Incumbent leader facing safety setbacks

Boys born with Duchenne muscular dystrophy typically lose the ability to walk before their teens and rarely live past 30. The first drugs designed to slow that decline reached the market in 2016, but they restore less than 1% of normal dystrophin, the muscle protein patients are missing. A decade later, a new wave of biotech companies is trying to deliver far more of that protein into muscle cells using engineered molecular shuttles.

Updated May 31

Gene therapy options for Duchenne muscular dystrophy

New Capabilities

Marketing Elevidys under restricted label

For three years, Sarepta Therapeutics has been the only company in the world selling a gene therapy for Duchenne muscular dystrophy. Regenxbio's trial result on May 14, 2026, sets up a second contender for U.S. Food and Drug Administration (FDA) approval.

Updated May 14