MDA Moves Newborn Screening Forward in Partnership with Cure SMA

This September as we recognize Newborn Screening Awareness month, we are proud to announce a new partnership with Cure SMA aimed at moving newborn screening forward for spinal muscular atrophy (SMA)—the leading genetic cause of death in infants. This is an exciting time for the neuromuscular disease community as decades of research are translating into . . .

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Catalyst Pharmaceuticals’ MG Drug Gets FDA Orphan Drug Designation

The investigational drug amifampridine phosphate (brand name Firdapse), under development by Catalyst Pharmaceuticals, has received U.S. Food and Drug Administration (FDA) Orphan Drug designation for the treatment of myasthenia gravis (MG). Amifampridine phosphate is a potassium channel inhibitor designed to prolong signals released from nerves and allow greater stimulation of muscles. The drug currently is . . .

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NIH Seeks Children and Caregivers to Participate in Quality of Life Study

Researchers at the National Institutes of Health (NIH) seek children with neuromuscular disorders (NMDs) and/or their caregivers (parent or legal guardian) to complete questionnaires. Researchers would like to develop a tool to better assess the quality of life of people with NMDs, such as muscular dystrophy. The goal of this study is to validate two different . . .

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Eteplirsen Granted Approval for DMD: Turning Promise into Progress

Today we couldn’t be happier as, together with the Duchenne community, we celebrate FDA accelerated approval of eteplirsen to treat kids and adults with some forms of Duchenne muscular dystrophy (DMD). “This is something that we’ve watched, and hoped and prayed for, and seen it develop over the last seven years or so,” said Josh . . .

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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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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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New Research Grants Aim to Help MDA Families Live Unlimited

  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 . . .

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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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