News

FDA investigates patient deaths after treatment with Sarepta's gene therapy
The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver ...
Pharmaceutical Technology1d
Sarepta and Roche halt DMD gene therapy’s use in certain patients after second death
Shares in both companies were down following news of the second death, casting fresh safety concerns over Elevidys.
Second patient death reported with gene therapy for muscular dystrophy
Shares of Sarepta Therapeutics have plunged again after the drugmaker reported a second death in connection with its gene ...
Sarepta Viral Vector Gets FDA Platform Technology Designation
Sarepta Therapeutics Inc. (NASDAQ:SRPT) is a commercial-stage biopharmaceutical company that discovers and develops ...
STAT5d
What’s next for Sarepta Therapeutics with gene therapy under fire?
The death of a boy with Duchenne muscular dystrophy, tied to a gene therapy, is above all a tragedy. But it is also ...
Rheumatology Advisor8h
Second Death Prompts New Safety Initiatives for DMD Gene Therapy Elevidys
Sarepta has issued a safety update regarding Elevidys (delandistrogene moxeparvovec-rokl) following a second reported case of acute liver failure resulting in death.
Two Children Dead After Gene Therapy: Florida Attorney Raises Alarms About FDA Oversight
A second child has died after receiving Elevidys, an experimental gene therapy for Duchenne muscular dystrophy—raising serious concerns about regulatory shortcuts, patient safety, and the urgent need ...
Sarepta Stock Plunges After Death of Second Patient Receiving Its Gene Therapy
A second patient receiving its experimental gene therapy Elevidys has died from liver failure, raising serious questions on ...
Sarepta reports second patient death after treatment with Duchenne gene therapy
Cambridge-based Sarepta Therapeutics said it was halting shipments of its Duchenne muscular dystrophy gene therapy for ...
Sarepta shares crash as second patient dies after receiving its gene therapy
Shares of Sarepta Therapeutics closed down as much as 42% to hit a nine-year low of $18.30 on Monday after a second death of ...
STAT5d
FDA’s gene therapy turmoil, and an alternative model for funding research
We look at the continued turmoil within the FDA, including the forced dismissal of top gene therapy regulator Nicole Verdun, ...
Sarepta Reports Second Death of Patient Using Its Gene Therapy
Sarepta Therapeutics Inc. said a second patient has died of acute liver failure after being treated with its gene therapy for ...