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 ...

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

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

While Sarepta has now consented to the FDA’s request to stop selling Elevidys, the company’s brief standoff with the agency ...

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

At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy, will carry a black box warning for acute liver injury and acute liver ...

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

REGENXBIO Inc. (NASDAQ:RGNX) is one of the biotech stocks to buy according to Wall Street analysts. On July 10, REGENXBIO ...

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