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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Encouraging Results in Nusinersen Trial for Infantile-Onset SMA

In a joint statement today, Biogen and Ionis Pharmaceuticals announced encouraging interim results from the late-stage ENDEAR clinical trial to test the experimental drug nusinersen in infants with type 1 spinal muscular atrophy (SMA). Based on the results, investigators have stopped the trial to allow all participants to transition into an open-label trial called SHINE, . . .

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Announcing MDA Amplify

MDA Amplify is a new mobile app that revolutionizes the way you — an MDA supporter, partner or family — can share MDA news on your own social media channels. To become an MDA Amplifier, download the MDA Amplify app from iTunes or Google Play store (it’s free!), connect your personal social media accounts, and . . .

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The Heart of Our Mission

At MDA, we are beginning 2016 with families at the heart of our mission – literally. The heart in the middle of our refreshed logo symbolizes the kids and adults living with muscular dystrophy, ALS and related life-threatening diseases we fight for every day. We also begin the year with new offerings and enhancements to . . .

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