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 . . .
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 . . .
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 (Radicava). Intravenous edaravone is an FDA-approved treatment for ALS shown to slow the decline of physical function. Edaravone is thought to act as a free . . .
Researchers at Scholar Rock are looking for individuals with spinal muscular atrophy (SMA) type 3 to participate in a phase 2 study. The goal of the study is to evaluate the safety and efficacy of SRK-015, which researchers hope may increase muscle mass and potentially improve muscle strength and motor function. The total study duration . . .
Yesterday, Mallinckrodt Pharmaceuticals released a statement to the Duchenne muscular dystrophy (DMD) community regarding its decision to discontinue its DMD clinical development program. The full statement follows. After careful consideration and despite engaging in diligent efforts to research, develop, and commercialize MNK-1411, Mallinckrodt has made the decision to terminate study MNK14112096 (BRAVE). The study was designed to . . .
Today, MDA announced new appointments to its board of directors. Steven J. Farella will now serve as chairman and Donald S. Wood, PhD, will serve as vice chairman of the MDA Board of Directors. “We are very pleased to share that both Steven Farella and Dr. Donald Wood have accepted their new positions on the . . .
Today, Solid Biosciences released a letter to the Duchenne muscular dystrophy (DMD) community regarding updates on a serious adverse event (SAE) experienced by one child dosed last November and new biomarker data from the company’s IGNITE DMD clinical trial. The trial is still on hold pending review by the US Food and Drug Administration (FDA). The full . . .
Today, Wave Life Sciences released a letter to the Duchenne muscular dystrophy (DMD) community regarding its decision to discontinue its DMD clinical development program for suvodirsen, its therapy for patients amenable to exon 51 skipping, and its therapy for patients amenable to exon 53 skipping. The full letter follows. Dear Duchenne community: We have a disappointing update . . .
MDA’s Medical Education department was established in response to the growing need for clinician education in neuromuscular disease (NMD). We are pleased to present four webinars that cover newborn screening (NBS) and early intervention, genetic testing, and best practices in multidisciplinary care. Our Medical Education department, together with key opinion leaders in the NMD field, . . .