News

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.

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 ability to climb up stairs should be assessed at intervals of nine months or longer and the ability to go down should be assessed annually in Duchenne muscular dystrophy (DMD) according to a new study.