Skip to Main Content

The Food and Drug Administration on Thursday approved Elevidys, Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy, for nearly all patients with the fatal muscle-wasting disease, dramatically expanding the initial, narrow authorization handed down last year.

The decision will likely be greeted by many families and doctors as a turning point in the fight against the muscle-wasting disease, even if it’s not nearly the seismic shift some had hoped for just a couple of years ago.


Gene therapy had long been seen as one of the approaches that might cure or at least halt the fatal condition. But clinical trials have presented a far murkier reality. Three different randomized studies of Duchenne gene therapies, including the only two conducted with Elevidys, have failed to reach their primary endpoints. And reports have circulated of boys — Duchenne mostly affects boys — whose conditions begin to decline just a couple years after treatment.

STAT+ Exclusive Story


This article is exclusive to STAT+ subscribers

Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.

Already have an account? Log in

Already have an account? Log in



Totals $468 per year

$39/month Get Started

Totals $468 per year



for 3 months, then $39/month

$30 for 3 months Get Started

Then $39/month



Save 15%

$399/year Get Started

Save 15%

11+ Users


Savings start at 25%!

Request A Quote Request A Quote

Savings start at 25%!

2-10 Users


Annually per user

$300/year Get Started

$300 Annually per user

View All Plans

Get unlimited access to award-winning journalism and exclusive events.


STAT encourages you to share your voice. We welcome your commentary, criticism, and expertise on our subscriber-only platform, STAT+ Connect

To submit a correction request, please visit our Contact Us page.