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Sarepta Therapeutics failed to win the European drug regulator's backing for its muscle disorder gene therapy on Friday, as ...
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.
The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.
European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...
Sarepta stock plunged again Thursday on a report that the Food and Drug Administration will require additional clinical ...