MDA Grants Work to Find Breakthroughs Across Diseases

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 . . .

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MDA Summer 2016 Research Grants [INFOGRAPHIC]

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. . . .

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Marathon Pharmaceuticals Announces Pivotal Phase 3 Data for Deflazacort for DMD in Neurology

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 . . .

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NDA Filing for ALS Treatment Accepted by FDA

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. . . .

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“MissionDMD” Trial Seeks Participants

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 . . .

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