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

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 ...

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

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

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 ...

New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular ...

A boy who was carried home from a birthday party suffering with what his parents thought was cramp has since been diagnosed ...

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

Sarepta Therapeutics said it has paused all shipments of its Elevidys treatment for Duchenne muscular dystrophy in the U.S.