Got questions? MDA’s new Resource Center has answers

MDA is pleased to announce that we have launched a new MDA National Resource Center that is now available to provide families one-on-one support from trained specialists. You can contact our MDA specialists at resourcecenter@mdausa.org and 1-833-ASK-MDA1 (1-833-275-6321) for resources and support. Specialists are available Monday through Friday 8 a.m. to 5:30 p.m. CST and are . . .

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Recent Treatment Triumphs Tied to MDA’s Long Commitment to Funding Treatments and Cures

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 . . .

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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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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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MDA-Supported Study Validates Surgery as a Treatment for Myasthenia Gravis

  In an MDA-supported worldwide study, researchers found that surgical removal of an organ called the thymus reduced muscle weakness and lowered the need for drugs that suppress the immune system in people with myasthenia gravis (MG). MG is an autoimmune neuromuscular disease that causes varying degrees of muscle weakness and fatigue. Initial treatment typically . . .

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FDA Will Review Deflazacort for Treatment of DMD

The U.S. Food and Drug Administration (FDA) has accepted Marathon Pharmaceuticals’ New Drug Applications for deflazacort for the treatment of Duchenne muscular dystrophy (DMD) and granted Priority Review. Deflazacort, a glucocorticoid, works as an anti-inflammatory and immunosuppressant. In the United States, the drug is considered an investigational therapy, as it has not been approved by . . .

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DMD Natural History Study Seeks Participants

Researchers are looking for boys and men, ages 5 to 18 years, to participate in a natural history study that is designed to assess the potential of imaging techniques to monitor disease progression and serve as an outcome measure for clinical trials in Duchenne muscular dystrophy (DMD). Both healthy volunteers and individuals with DMD are . . .

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