News

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 ...
New consensus guidelines outline best practices for orthopedic management in Duchenne muscular dystrophy, focusing on ...
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 ...
Duchenne muscular dystrophy families are sharing stories about Elevidys complications on social media. But they aren’t ...
Sarepta Therapeutics Inc. misled investors about the safety and prospects for one of its Duchenne muscular dystrophy ...
The FDA found that data from a single Phase II study were “insufficient” to justify an accelerated approval review for ...
In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...
Edgewise Therapeutics (NASDAQ:EWTX) lost ~9% in the morning hours on Thursday after the company posted data from clinical ...
The FDA said yesterday that it is investigating the deaths from liver failure of two non-ambulatory boys with Duchenne ...
The MESA data demonstrated sustained disease stabilization, reinforcing prior ARCH and CANYON findings. Importantly, CANYON ...
Edgewise reports sustained functional stability in Becker patients and selects Phase 3 dose for Duchenne after positive Phase ...
FDA has indicated that an Advisory Committee meeting is not required at this timeIn-person late-cycle review meeting ...