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

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

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 ...

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

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 ...