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

Absent a federal committee’s recommendation, Duchenne muscular dystrophy advocates are trying to get states to adopt ...

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

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

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

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

Edgewise Therapeutics (NASDAQ:EWTX) lost ~9% in the morning hours on Thursday after the company posted data from clinical ...

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 ...

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 ...