News

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 ...
At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...
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 Sarepta saga continues, with the FDA slapping a clinical hold across all of the company’s investigational limb girdle ...
As mothers of children with this disease, we have wept helplessly in recent months as friends — fellow members of a club we ...
The first patient has been dosed in a clinical trial testing Sardocor's one-time gene therapy for cardiomyopathy associated ...