Archives

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…

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…

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…

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,…

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…

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 —…

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…

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…

Researchers at Mitsubishi Tanabe Pharma Development America are looking for participants with amyotrophic lateral sclerosis (ALS) to participate in a phase 3 study to evaluate the safety of oral edaravone…

On Jan. 22, Genentech, a member of the Roche Group, announced positive results from the second part of its pivotal phase 2/3 FIREFISH clinical trial assessing risdiplam in infants age…