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

The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.

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

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.

Sarepta Therapeutics failed to win the European drug regulator's backing for its muscle disorder gene therapy on Friday, as ...

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

Sarepta Therapeutics (NASDAQ:SRPT) faces an “arduous and treacherous path” to bring its gene therapy Elevidys back to market, ...

Duchenne Muscular Dystrophy affects 12,000 to 15,000 children and young adults in the United States and about 300,000 worldwide. It's caused by a mutation in the dystrophin gene, which makes a ...

Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...

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