Skip to Main Content
Contribute Try STAT+ Today

The Food and Drug Administration on Thursday approved a new medicine from Sarepta Therapeutics that treats certain patients with Duchenne muscular dystrophy.

The new drug, called Amondys 45, is Sarepta’s third marketed treatment for the rare, inherited muscle-wasting disease. It will be used to treat the approximately 8% of patients with a confirmed mutation to the Duchenne gene that is amenable to exon 45 skipping.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!


What is it?

STAT+ is STAT's premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond.

What's included?

  • Daily reporting and analysis
  • The most comprehensive industry coverage from a powerhouse team of reporters
  • Subscriber-only newsletters
  • Daily newsletters to brief you on the most important industry news of the day
  • STAT+ Conversations
  • Weekly opportunities to engage with our reporters and leading industry experts in live video conversations
  • Exclusive industry events
  • Premium access to subscriber-only networking events around the country
  • The best reporters in the industry
  • The most trusted and well-connected newsroom in the health care industry
  • And much more
  • Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr.
  • I’m a person with ALS and just this week the FDA did not approve NurOwn for treatment of ALS. Interesting how the drugs above have been fast tracked through the FDA and yet ALS has seen nothing for new drugs being approved.

    Adam we pALS would love to see you investigate why NurOwn and other such drugs with much better results are not being appoved by the FDA and based on what you’ve written above should all be out there for us pALS to use.

    We need your help!


Comments are closed.