The biotechnology company Sarepta Therapeutics has decided to suspend entirely one of its treatments for Duchenne muscular dystrophy (DMD) just days after it refused a request from the US Food and Drug Administration (FDA) to pull the drug from the market.

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 failure, drugmaker Sarepta Therapeutics said.

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 ...

Melanie Sanford fought to get her son Hudson a breakthrough gene therapy to stop the progression of the fatal disease Duchenne Muscular Dystrophy. She rushed to get him access just days before he ...

The Sarepta saga continues, with the FDA slapping a clinical hold across all of the company’s investigational limb girdle muscular dystrophy (LGMD) trials, while also revoking the biopharma’s gene therapy platform technology designation.

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.

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