News

Fierce Pharma1h
FDA investigates patient deaths after treatment with Sarepta's Duchenne gene therapy Elevidys
In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...
STAT2h
Pharmalittle: We’re reading about FDA probing deaths after Sarepta treatment, a Regeneron plan for charities, and more
The FDA is investigating two deaths in non-ambulatory Duchenne muscular dystrophy patients who received a Sarepta gene ...
MedPage Today2h
FDA Probes Gene Therapy After Two Deaths
Duchenne muscular dystrophy is characterized by a mutation in the DMD gene that leads to a lack of dystrophin and muscle loss ...
Devdiscourse3h
U.S. Domestic Briefs: Health Challenges, Tech Advancements, and Political Upsets
Recent U.S. domestic news covers the FDA investigating deaths post-gene therapy by Sarepta, discussions at a CDC advisory ...
BioSpace3h
FDA Reviews Sarepta’s Elevidys After Second Patient Death
The FDA is assessing the need for “further regulatory action” on Sarepta's Duchenne muscular dystrophy gene therapy in the ...
pharmaphorum4h
FDA probing deaths with DMD gene therapy Elevidys
Elevidys has been given full approval to treat ambulatory patients with DMD, with an accelerated approval in non-ambulatory ...
Devdiscourse16h
Key Rulings & Measures Shape US Economic and Regulatory Landscape
Recent developments in U.S. domestic affairs include a legal victory for Anthropic in an AI training lawsuit, Treasury ...
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 ...
Sarepta Viral Vector Gets FDA Platform Technology Designation
Sarepta Therapeutics Inc. (NASDAQ:SRPT) is a commercial-stage biopharmaceutical company that discovers and develops ...
Rheumatology Advisor1d
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.