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.

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

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

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

European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...

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

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

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

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

Despite a new setback for Elevidys in Europe, Roche—which markets Sarepta’s gene therapy outside the U.S.—remains committed ...

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 ...