Contribute Try STAT+ Today

Sarepta Therapeutics said Monday that three children born with a rare muscle-wasting disease called limb-girdle muscular dystrophy are showing durable improvements in strength and function a year after a one-time infusion of an experimental gene therapy.

Three more children have been treated with a higher dose of the same gene therapy. Initial biopsy results from those patients, also reported Monday, show greater production of a key muscle protein with no added safety problems — suggesting the higher dose could lead to even larger improvements in muscle function. Those tests will be performed later this year, Sarepta said.

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.

Comments are closed.