Sarepta Therapeutics

Stories

Gene therapy options for Duchenne muscular dystrophy

Marketing Elevidys under restricted label

Regenxbio's RGX-202 met the main goal of its AFFINITY DUCHENNE trial in May 2026. In 93% of treated boys with usable biopsies, muscle made microdystrophin, a shortened form of the missing protein, at more than 10% of normal levels by week 12. The company finished dosing a confirmatory study and plans to file for FDA approval in the third quarter of 2026.

Updated Nov 1

The race to build a better Duchenne therapy

New Capabilities

As of May 2026: 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