We know families across the country, especially those in the neuromuscular disease (NMD) community, are concerned about the spread of coronavirus and its associated disease, COVID-19, and the impact on their loved ones. In these uncertain times, MDA’s advocacy team will not stop serving and amplifying the voices of those in the NMD community, especially . . .
On March 9, Acceleron Pharma, Inc., announced that a phase 2 clinical trial of its therapy under development, ACE-083, for the treatment of Charcot-Marie-Tooth disease (CMT) did not show statistically significant improvements in functional tests compared to placebo. As a result, Acceleron is discontinuing development of ACE-083 and its clinical trials program for CMT. Delivered by . . .
On March 9, NS Pharma Inc. (a wholly owned US subsidiary of Nippon Shinyaku Co. Ltd.) announced that it has launched an Expanded Access Program in the United States for viltolarsen, its investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. The program is open to all DMD patients . . .
Since 2011, MDA Muscle Walks have been gathering individuals living with muscular dystrophy, ALS, and related neuromuscular diseases with their families, friends, and communities across America. Together, they’ve raised awareness of everyday life with muscle disease — and they’ve raised $49 million to fund expert care and innovative research directed toward delivering cures. This year, . . .
Myopathy is a disease condition where muscle fibers do not function properly, resulting in muscular weakness. Inclusion-body myositis (IBM) is a type of inflammatory myopathy that is characterized by inflammation, weakness, and atrophy of the skeletal muscle. IBM develops in adulthood, usually after age 50. The symptoms and rate of progression vary from person to . . .
Researchers at Virginia Commonwealth University are seeking participants living with limb-girdle muscular dystrophy type 2I (LGMD2I) to participate in a natural history study. This study aims to better understand disease course so researchers will be able to determine clinically meaningful outcome measures for use in future clinical trials, including the utility of dystroglycan as a biomarker. This two-year . . .
On Feb. 7, NS Pharma Inc. (a wholly owned US subsidiary of Nippon Shinyaku Co. Ltd.), announced that the US Food and Drug Administration (FDA) accepted its New Drug Application (NDA) seeking approval under Priority Review for viltolarsen, an investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. Priority . . .
Last year, Sharon Hesterlee, PhD, took the helm as executive vice president, chief research officer, at MDA. With her unique, decades-spanning career — which began, in fact, at MDA — in the pharmaceutical, biotechnology, and nonprofit sectors, her appointment is certain to advance not only MDA’s mission but also the research and development of genetic . . .
Researchers at Expansion Therapeutics are looking for adults with myotonic dystrophy type 1 (DM1) to participate in a phase 1 study. The goal of the study is to evaluate the safety of ERX-963. Researchers hope this therapy may improve symptoms of excessive daytime sleepiness and improve attention and memory. All participants will receive one dose . . .
Researchers at PTC Therapeutics are looking for individuals with limb-girdle muscular dystrophy type 2I (LGMD2I) to participate in a phase 3 study. The goal of the study is to evaluate the efficacy of deflazacort (Emflaza), which researchers hope may reduce inflammation in muscles and potentially lead to improved muscle strength and motor function. Participants will . . .