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The biotech, which cut jobs following the rejection in May of its Barth syndrome therapy, claimed it has addressed the agency ...

Sarepta Therapeutics has provided additional safety data on its gene therapy Elevidys as a Duchenne muscular dystrophy ...

The deaths prompted the FDA to ask Sarepta to stop shipping Elevidys for all patients, but the company refused to do so. Days later, it changed tack in order to maintain what it said was a “positive ...

University of Manchester scientists have mapped the mutations in the tiny protein chains that cause a subtype of muscular ...

Starting oral risdiplam soon after birth may put babies with spinal muscular atrophy on the path to normal development, new ...

The Health Resources and Services Administration is requesting public comments on its recommendation to add metachromatic ...

Arrowhead Pharmaceuticals stock tumbled Thursday after Sarepta Therapeutics liquefied its entire stake in the smaller biotech ...

White House chief of staff Susie Wiles intervened to save the job of a top health official President Donald Trump had fired ...

Liquidating its stake in Arrowhead helps Sarepta cover a milestone payment and secure what one analyst described as ...

Sarepta will sell back $50 million worth of shares to cover part of a $100 million milestone payment and its remaining shares worth $174 million.

After two patients who received the investigational CDC7 blocker died, pushing forward with SGR-2921’s development would be ...