The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...

The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne ...

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...

Genetic testing could not confirm Duchenne muscular dystrophy, but a muscle biopsy at UCLA did. "He could see that he was ...

Basel: Roche has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency ...

EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory ...

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 ...

The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.

The FDA is investigating the death last month of an eight-year-old boy in Brazil who had received Sarepta Therapeutics’ Elevidys® (delandistrogene moxeparvovec-rokl)—though the company and its ...

The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could ...

Shipments will halt by close of business Tuesday evening, the company said. Sarepta had initially rejected the agency’s ...