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FDA weighs new study requirements for Sarepta to confirm safety of gene therapy Elevidys: report
Sarepta would need to conduct new analyses to validate the safety of Elevidys—which has had U.S. shipments paused by the company over a mounting string of controversies—to the FDA, Endpoints News and Bloomberg separately reported Thursday, citing an unnamed senior FDA official.
The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients died, the drug’s manufacturer halted dosing under pressure from the FDA.
Drugmaker Sarepta Therapeutics says it won’t comply with a request from U.S. regulators to halt all shipments of its gene ...
Sarepta Therapeutics’ Duchenne therapy faces ‘arduous’ path back to market, senior FDA official says
Sarepta Therapeutics faces an "arduous path” to try to get its gene therapy for Duchenne muscular dystrophy back onto the market, a senior FDA official told STAT.
The FDA has placed multiple investigational gene therapy clinical trials on hold, signaling broader platform concerns.
Sarepta Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that ...
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