The U.S. FDA has corresponded with Santhera Pharmaceuticals regarding Santhera’s proposed subpart H approval pathway of Raxone (idebenone) in patients with…
Articles by Margarida Azevedo, MSc
Catabasis Pharmaceuticals has initiated an open-label extension of the Phase 2 portion (Part B) of the MoveDMD clinical trial investigating…
Capricor Therapeutics has reached half its target enrollment for its ongoing HOPE-Duchenne clinical trial, the company announced. Capricor discovers, develops, and…
Two annual fundraisers – the 2016 Appetite for a Cure and the Annual MDA Golf Classic – raised a recordbreaking $838,358…
The American Academy of Neurology (AAN), together with the American Brain Foundation (ABF) and the Muscular Dystrophy…
PTC Therapeutics has successfully negotiated a Managed Access Agreement (MAA) with the National Health Service (NHS) in England for…
Acceleron Pharma will present the results of a Phase 1 clinical study of ACE-083, its lead investigational therapeutic for patients…
MaestroSoft, a fundraising solutions company, announced that its Text2Bid technology was used to support the Muscular Dystrophy’s Association of…
Parent Project Muscular Dystrophy (PPMD), a non-profit organization fighting to find a cure for Duchenne muscular dystrophy (DMD),…
The FSH Society, a non-profit organization led by facioscapulohumeral muscular dystrophy (FSHD) patients in support of research and education,…