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

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

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

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

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

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

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

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

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...

The U.S. Food and Drug Administration said on Friday that it is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta Therapeutics ( NASDAQ: SRPT) gene therapy for Duchenne ...

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

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