Catalyst Pharmaceuticals, Inc.

Rare and pediatric disease drug sponsors and the US Food and Drug Administration could resolve several ongoing rare and pediatric drug development issues in a compromise bill to fund the government th

Santhera Pharmaceuticals says that its Duchenne muscular dystrophy (DMD) treatment Agamree has got off to a strong start and while pricing negotiations are unsurprisingly proving tricky, the Swiss fi

The US Food and Drug Administration’s full approval of Sarepta Therapeutics, Inc. ’s Elevidys (delandistrogene moxeparvovec-rokl) pastes another jewel in the company’s “most contentious approvals” cr

In a recent discussion forum on China's WeChat social media platform, a group of patients whose sons are suffering from Duchenne muscular dystrophy (DMD) had good news to share - neurologists at one o