Potential Tool to Test MD Therapies: Lab-grown Muscle Fibers That Stay Alive Longer with New Gelatin Chip
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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Phase 1 Trials Results for FSHD Therapy to Be Presented at Neuromuscular Congress
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…
Maestro’s Text2Bid Helps MDA Chapter Raise $100,000 in Silent Auction at Black-N-Blue Ball
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 Receives Marathon Pharmaceuticals Grant for Duchenne Centers
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…
Ability of Duchenne MD Children to Use Stairs Should Be Evaluated Every 9-12 Months, Study Suggests
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.
Facioscapulohumeral Muscular Dystrophy Gets First World Awareness Day
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 to Present Data from MoveDMD Trial in Duchenne Patients at PPMD 2016 Annual Conference
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…
Drug to Possibly Treat DMD, Idebenone (Raxone), Under CHMP Review in Europe
Santhera Pharmaceuticals reports that the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) for its drug Raxone (idebenone; U.S. brand name, Catena) as a treatment for Duchenne muscular dystrophy (DMD) in patients with respiratory function decline and who are not under glucocorticoid therapy. The EMA validation confirms…
UC San Diego Grad with MD Won’t Stop Fighting for His Dreams
Raul Carranza was born in Chula Vista 27 years ago. The recent graduate in political science from the University of California San Diego, and future law student, is one of the university’s most heralded students. Diagnosed with muscular dystrophy (MD) at the age of two, and by his third birthday…
Heart Arrhythmia in Myotonic Dystrophy Patients Traced to Mutant RNA in Study
New research suggests that heart arrhythmia in myotonic dystrophy (DM) patients is caused by mutant RNA in the heart’s sodium channel. The study, which adds to the understanding of this symptom, was titled “Splicing misregulation of SCN5A contributes to cardiac-conduction delay and heart arrhythmia in myotonic dystrophy,” and published in …
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