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

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

Three patient deaths linked to neuromuscular drugs developed by Sarepta Therapeutics (NASDAQ:SRPT) have drawn regulatory ...

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 individuals with Duchenne muscular dystrophy (DMD) Roche will continue its ...

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

The three deaths resulted from acute liver failure in patients treated with either Elevidys or investigational therapies using the same AAVrh74 viral vector technology. One fatality occurred during a ...

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

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

The company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still ...

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 ...