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

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

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

A mouse model study of Precision BioSciences’ gene-editing therapy PBGENE-DMD found it produced long-term functional ...

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.

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

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

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

A new pioneering gene therapy Elevidys works by replacing the Duchenne variant of dystrophin with man-made version based on a version found in a patient with a milder form of the disease, called ...

The study aims to evaluate the safety and efficacy of a gene therapy, PF-06939926, in boys with Duchenne Muscular Dystrophy (DMD), a severe genetic disorder characterized by progressive muscle ...

WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy.