An alternative to using standard glucocorticoids is the synthetic dissociative steroid vamorolone (Agamree), which was ...

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

The Food and Drug Administration has paused distribution of the gene therapy Elevidys following the death of a third patient ...

Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver ...

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...

Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...

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

Genethon is determined to bring GNT0004 to market for young patients and their families who are waiting for a therapeutic ...

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

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

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