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Medpage Today on MSN7h
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 ...
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 ...
Fierce Pharma7h
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, ...
FDA probes deaths of boys taking Sarepta Duchenne drug
The FDA said yesterday that it is investigating the deaths from liver failure of two non-ambulatory boys with Duchenne ...
BioSpace8h
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 ...
pharmaphorum9h
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 ...
STAT7h
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 ...
Did The FDA Make A Mistake? Sarepta's Elevidys Approval Under Scrutiny
FDA is investigating Sarepta's Elevidys after two deaths from liver failure in non-ambulatory DMD patients, raising safety ...
Devdiscourse8h
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 ...
Devdiscourse21h
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 ...
Fintel on MSN9h
HC Wainwright & Co. Downgrades Sarepta Therapeutics (SRPT)
Fintel reports that on June 25, 2025, HC Wainwright & Co. downgraded their outlook for Sarepta Therapeutics (NasdaqGS:SRPT) ...
Medindia16h
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 ...