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…
News
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…
A newly published study, “Enhancement of Satellite Cell Transplantation Efficiency by Leukemia Inhibitory Factor,” indicates that the cells, also known as muscle stem cells, treated with leukemia inhibitory factor (LIF) are better at forming new muscle fibers when transplanted into the body Satellite cell transplantation has been studied as a therapeutic approach…
An international team of researchers has reviewed the latest treatments targeting non-muscle cells in the heart responsible for cardiac scarring. The information is important for developing strategies to treat diseases that are often viewed as hopeless, from common ailments like advanced heart failure to rare but deadly ones like muscular dystrophy. The study from…
Scientists developed a new gelatin matrix or “chip”, which allows them to grow muscle fibers in the laboratory for at least three weeks.
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