Margarida Azevedo, MSc,  —

The U.S. FDA has corresponded with Santhera Pharmaceuticals regarding Santhera’s proposed subpart H approval pathway of Raxone (idebenone) in patients with Duchenne muscular dystrophy (DMD) who are not taking concomitant glucocorticoids. Santhera had proposed that results of the SIDEROS clinical trial provide confirmatory evidence of efficacy in these patients while…

Catabasis Pharmaceuticals has initiated an open-label extension of the Phase 2 portion (Part B) of the MoveDMD clinical trial investigating edasalonexent (CAT-1004) in the treatment of boys with Duchenne muscular dystrophy (DMD). Edasalonexent is a non-corticosteroid, oral small molecule investigational drug that inhibits activated NF-ĸB. In boys with Duchenne MD, the…

Capricor Therapeutics has reached half its target enrollment for its ongoing HOPE-Duchenne clinical trial, the company announced. Capricor discovers, develops, and commercializes first-in-class therapeutics such as the cardiac cell therapy (CAP-1002) for the treatment of Duchenne muscular dystrophy (DMD)-associated cardiomyopathy. The company expects to complete enrollment by the end of…

Two annual fundraisers – the 2016 Appetite for a Cure and the Annual MDA Golf Classic – raised a recordbreaking $838,358 combined for children and adults suffering from muscular dystrophy (MD) and related muscle-degenerating diseases. The fundraisers were organized and carried out by by Sailormen Inc., one of the biggest national…

The American Academy of Neurology (AAN), together with the American Brain Foundation (ABF) and the Muscular Dystrophy Association (MDA), are partnering to offer a new Clinical Research Training Fellowship in muscular dystrophy (MD) starting in 2017. Because of the serious need for continuous neurology research, the AAN…

PTC Therapeutics has successfully negotiated a Managed Access Agreement (MAA) with the National Health Service (NHS) in England for Translarna (ataluren) to treat ambulatory patients ages 5 and older with nonsense mutation Duchenne muscular dystrophy (nmDMD). The decision provides reimbursed patient access to Translarna in England via a five-year…

Acceleron Pharma will present the results of a Phase 1 clinical study of ACE-083, its lead investigational therapeutic for patients with facioscapulohumeral muscular dystrophy, at the 14th International Congress on Neuromuscular Disease (ICNMD 2016) taking place in Toronto, Canada, on July 5–9. According to a press release, the poster, “ACE-083, A Locally-Acting…

MaestroSoft, a fundraising solutions company, announced that its Text2Bid technology was used to support the Muscular Dystrophy’s Association of Southern Wisconsin’s Annual Black-N-Blue Ball, a money-raising event for muscular dystrophy (MD). Text2Bid allows guests at auctions to be notified by text message on the status of their bidding, making…

Parent Project Muscular Dystrophy (PPMD), a non-profit organization fighting to find a cure for Duchenne muscular dystrophy (DMD), recently announced that Marathon Pharmaceuticals will support the organization’s ongoing Certified Duchenne Care Center (CDCC) Program with a new grant. “We recognize that the Duchenne community needs the kind of…

The FSH Society, a non-profit organization led by facioscapulohumeral muscular dystrophy (FSHD) patients in support of research and education, joined other FSH groups worldwide in hosting the first World FSHD Day on June 20. The day was developed with FSHD Champions, an international alliance of organizations advocating for awareness…