The family of a seven-year-old boy diagnosed with a life-changing condition are raising funds to build a specially-adapted ...

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

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

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

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

US regulators are investigating the death of an 8-year-old boy in Brazil who received Sarepta Therapeutics Inc.’s Elevidys.

The patient with Duchenne muscular dystrophy, who died in Brazil, was treated with Elevidys but was not a participant in a ...

EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory individuals with Duchenne muscular dystrophy (DMD) Roche will continue its ...

The U.S. Food and Drug Administration (FDA) is investigating the death of an eight-year-old boy who received Sarepta ...

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

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

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