News

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.

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…

Catabasis’ chief medical officer, Dr. Joanne Donovan, will present results from the first phase of the two-part MoveDMD Trial, exploring the company’s investigational drug edasalonexent (CAT-1004) in patients with Duchenne muscular dystrophy (DMD). The presentation will take place at the 2016 Parent Project Muscular Dystrophy (PPMD) Annual Connect Conference, set…