As mothers of children with this disease, we have wept helplessly in recent months as friends — fellow members of a club we never asked to join — said goodbye to their sons, the babies they once held in their arms, whose dreams they held in their hearts until Duchenne robbed them of working muscles or a healthy future.

Shares of Sarepta Therapeutics plunged more than 30% on Friday as the future of its approved gene therapy appeared at risk. Sarepta has reported three patient deaths related to its gene therapies.

Duchenne Muscular Dystrophy affects 12,000 to 15,000 children and young adults in the United States and about 300,000 worldwide. It's caused by a mutation in the dystrophin gene, which makes a ...

The first patient has been dosed in a clinical trial testing Sardocor's one-time gene therapy for cardiomyopathy associated with Duchenne MD.

Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta Therapeutics adds a black box warning to the gene therapy for acute liver injury and failure and parts with more than a third of employees.

Sarepta Therapeutics announced it has laid off more than one-third of its workforce, a drastic cost-cutting move following the deaths of two teenagers administered its gene therapy for Duchenne muscular dystrophy.

Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders including Duchenne muscular dystrophy,