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More than 800 people have been treated with Elevidys, the only approved treatment for Duchenne muscular dystrophy, the ...
Sarepta Therapeutics said on Tuesday that a 16-year-old boy died from acute liver failure months after receiving the company's U.S-approved gene therapy for a rare muscular dystrophy.
Mizuho says that while “any clinical hold is concerning,” the European Medicines Agency’s request to pause the three Elevidys studies will ...