Two patients in a study of a high-dose gene therapy for a rare muscle disorder have died, heightening worries about the safety of potent treatments under development for other diseases.

Audentes Therapeutics, a San Francisco-based unit of the Japanese drug firm Astellas, told patient groups in a June 23 letter, a copy of which was obtained by STAT, that the two patients died after having serious liver problems that appeared related to the company’s experimental treatment for X-linked myotubular myopathy, a condition that causes severe muscle weakness. Most patients with the disorder, also known as XLMTM, do not survive childhood. In previous data from the study, Audentes has said its treatment improved patients’ ability to breathe, with some getting off of ventilators.

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

GET STARTED

What is it?

STAT Plus 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
  • Online intelligence briefings
  • Frequent opportunities to engage with veteran beat reporters and industry experts
  • 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.

A roundup of STAT’s top stories of the day in science and medicine

Privacy Policy