Breaking: Second patient death is reported by Sarepta after Duchenne gene therapy

Both occurred from acute liver failure, a side effect that has been seen with other gene therapies.

Log In    Subscribe BREAKING NEWS   Sarepta reports second patient death after treatment with Duchenne gene therapy By Andrew Joseph Kristoffer Tripplaar/Sipa via AP Sarepta Therapeutics said Sunday that it was halting shipments of its Duchenne muscular dystrophy gene therapy for patients who can no longer walk, following the death of a second person who received the treatment. Sarepta disclosed the first patient death — a 16-year-old boy — in March. Both occurred from acute liver failure, a side effect that has been seen with other gene therapies.   The death is certain to add additional scrutiny to the FDA's decision last year to authorize the therapy so broadly, even with limited data in older patients. READ MORE Be the first to know Get instant notifications for the topics and reporters you choose with STAT+ Email Alerts. In Case You Missed It 1   FDA reassures rare disease advocates that 'being flexible' is plan for gene therapy By Jason Mast 2   Sarepta demanded Duchenne patient advocacy group censor video critical of the company By Adam Feuerstein 3   Trump administration demands pharma companies begin drug price negotiations, a day after key deadline By Daniel Payne 4   Recursion CEO: Biotech's rainy season is not over — but we must race ahead By Chris Gibson Get 50% off STAT+ Stay up to date on the latest news coming out of the biotech, pharma, and life science industries. Learn more STAT, 1 Exchange Place, Boston, MA ©2025, All Rights Reserved. I no longer wish to receive STAT emails Update Newsletter Preferences • Contact Us • Advertise with Us