The National Institute for Health and Care Excellence (NICE) has just issued new guidance recommending Translarna (ataluren) for children older than 5 who have Duchenne muscular dystrophy (DMD). The guidance, titled “Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene,” follows the…
Duchenne UK is launching a new video fundraising and awareness campaign for Duchenne muscular dystrophy (DMD) called ‘The World’s Strongest Boys’. The campaign is already supported by British sports celebrities including Jessica Ennis-Hill and Owen Farrell to help boys living with DMD think like heroes by focusing on…
For the third consecutive year, the Muscular Dystrophy Association‘s nationwide endurance program, the MDA Team Momentum, is revving to run in the Dec. 11, 2016 BMW Dallas Marathon, Half Marathon and Behringer Relay . So far, more than 150 participants on 61 registered teams are geared up already for MDA – even before registrations begins. Since 2014, more…
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…
PLX (placental expanded)-PAD cells, a line of pluripotent stem cells produced promising results in a mouse model of Duchenne muscular dystrophy.
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…
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