Henry Houlden, professor of neurology at the MRC Centre for Neuromuscular Diseases, University College London Institute of Neurology in England, was awarded an MDA research grant totaling $288,151 over three years to elucidate the genetic causes of severe forms of Charcot-Marie-Tooth disease (CMT) and other types of early-onset neuropathy. Identification of the causative genes could . . .
Over the last six decades, MDA has committed $1 billion to research designed to move the world closer to treatments and cures for muscular dystrophy, ALS and related life-threatening diseases. As a result, MDA’s fingerprints are on nearly every major neuromuscular disease research breakthrough, and the progress we continue to see in the field is . . .
Rachelle Crosbie-Watson, professor and vice-chair of graduate education at the University of California Los Angeles, was awarded an MDA research grant this summer totaling $300,000 over a period of three years to test new therapies for cardiomyopathy (heart muscle weakness) associated with Duchenne muscular dystrophy (DMD). Can you tell us a little bit about your . . .
Since its inception, MDA has invested more than $1 billion in neuromuscular disease research to uncover new treatments and cures. In 2016 alone, MDA awarded 66 new research, development and research infrastructure grants with a total funding commitment of more than $17 million. These and other MDA grants fund research projects designed to uncover the . . .
This summer MDA challenged our families, friends, sponsors, staff and others around the nation to share their stories about living life without limits despite the challenges of neuromuscular disease. Some shared moments tied to pursuing an education or career that was thought to be out of reach. Some talked about skydiving, hiking or running a . . .
Groundbreaking research plays a pivotal role in our mission to accelerate treatments and cures for muscular dystrophy, ALS and related life-threatening diseases. Each winter and summer, we award grants to researchers worldwide studying to make impacts across diseases. For our Summer 2016 Research Grants Cycle, we’re happy to announce $6.7 million in newly awarded funding. . . .
Marathon Pharmaceuticals has announced pivotal Phase 3 data evaluating the investigational drug deflazacort for the treatment of Duchenne muscular dystrophy in the journal Neurology. The study results show that deflazacort demonstrated significant improvement in muscle strength compared with placebo in 12 weeks. In addition, results show that deflazacort was associated with less weight gain than . . .
Osaka-based Mitsubishi Tanabe Pharma announced today that its New Drug Application (NDA) for the drug edaravone to treat people with ALS (amyotrophic lateral sclerosis) was accepted by the U.S. Food and Drug Administration. The Japanese pharmaceutical company said it expects a decision on whether it can market the drug in the United States by June 16, 2017. . . .
Researchers are looking for boys and men living with Duchenne muscular dystrophy, ages 12 years and older, to participate in a phase 2 clinical trial, sponsored by FibroGen, to test the experimental drug FG-3019 (pamrevlumab). The study, known as “MissionDMD,” is designed to help researchers evaluate whether FG-3019 is safe and effective in people with . . .
Every August is Spinal Muscular Atrophy Awareness Month, and this year we have a lot to celebrate. At the beginning of August, Biogen and IONIS Pharmaceuticals announced encouraging results from a late-stage clinical trial testing an experimental drug for infants with SMA type 1. The drug is called nusinersen and is designed to increase production . . .
