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 . . .
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 firstname.lastname@example.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 . . .
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 . . .
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 . . .
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 . . .
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 . . .
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 . . .