Dysfunctional dystrophin, the muscle protein affected in Duchenne, leads to an important choice for patients about how to ...

New consensus guidelines outline best practices for orthopedic management in Duchenne muscular dystrophy, focusing on ...

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...

Sarepta Therapeutics Inc. misled investors about the safety and prospects for one of its Duchenne muscular dystrophy ...

The FDA said yesterday that it is investigating the deaths from liver failure of two non-ambulatory boys with Duchenne ...

One visit to Virginia Peninsula Community College’s Historic Triangle Campus last summer eased any concerns they might have had. Soon after entering the building, Nicholas and his mother, Liz, were ...

The FDA found that data from a single Phase II study were “insufficient” to justify an accelerated approval review for ...

Shares of Sarepta Therapeutics have plunged again after the drugmaker reported a second death in connection with its gene ...

The association of Duchenne muscular dystrophy with another central nervous system disorder, autism spectrum disorder/pervasive developmental disorder, which we describe, ...

Duchenne muscular dystrophy patient died after being treated with the gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta Therapeutics said Sunday. Like the first case reported, this patient ...

Capricor's DMD therapy Deramiocel is under FDA Priority Review with no major issues flagged; PDUFA date set for August 31, ...

The drug Givinostat has been brought into parts of Britain. We want that too, but we need the government to pay for it,' said Una Ennis ...