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Sarepta Therapeutics said the Food and Drug Administration informed the company it can lift a voluntary pause on shipments of Elevidys for ambulatory patients with Duchenne. The company said Monday ...
The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.
The U.S. Food and Drug Administration said on Friday it was investigating the death of an eight-year-old boy who received ...
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The story of Sarepta's Duchenne gene therapy
The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...
Europe's medicines regulator did not recommend the approval of Sarepta Therapeutics' gene therapy Elevidys for a type of rare ...