Encouraging Clinical Study Data Reported for ALS Drug Edaravone

Osaka-based Mitsubishi Tanabe Pharma has reported encouraging 12-month efficacy and safety data for edaravone (brand name Radicava) for the treatment of ALS. Edaravone is delivered by intravenous injection. It’s thought to work by relieving the effects of oxidative stress, which, in people with ALS, has been suspected to play a role in the death of . . .

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MDA Holiday Gift Guide

Few things are better than the perfect gift — except, perhaps, the gift that gives love to the recipient and to a whole community as well. This season, surprise your family with gifts that give to MDA families — and MDA research — too. Good Reads Stack your family bookshelf with must-reads that help explain . . .

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MDA and RYR-1 Foundation Team Up in the Fight Against RYR1-Related Myopathies

MDA and RYR-1 Foundation have announced a partnership aimed at advancing research and clinical care, raising awareness and improving education of patients, medical professionals and the public about RYR1-related myopathies. The partnership represents a key step in MDA’s commitment to form collaborative relationships with other organizations working on the same diseases MDA covers. “MDA is . . .

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Meet the MDA Resource Specialists: Mandy

MDA Resource CenterMDA resource specialist Mandy Behling used to think that living unlimited meant swimming with the sharks and climbing Mt. Everest. However, she has come to find that living unlimited means more than just traveling the world and seeking out grandiose adventures.

“Now I think that living unlimited means pushing myself just a little harder than I thought possible the day before,” she says.

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Meet Ashleigh: MDA Resource Specialist

If there was one piece of advice MDA Resource Specialist Ashleigh Peska could offer individuals with muscular dystrophy and other muscle-debilitating diseases, it would simply be, “don’t quit!” Ashleigh, who lives with limb-girdle muscular dystrophy (LGMD), is a shining example of this mantra. A college graduate and former MDA Iowa State Goodwill Ambassador, Ashleigh has . . .

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Top 5 FAQs: Eteplirsen (Exondys 51) for DMD Treatment

On Sept. 19, the U.S. Food and Drug Administration granted accelerated approval to eteplirsen, which will now be marketed under the brand name Exondys 51 for the treatment of some forms of Duchenne muscular dystrophy (DMD). We’ve received a lot of questions about the newly approved drug, and wanted to share a few answers to . . .

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Biogen Submits New Drug Application for Nusinersen to Become an Approved Treatment for SMA

In a joint statement today, Biogen and Ionis Pharmaceuticals announced that Biogen has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen, an investigational treatment for spinal muscular atrophy (SMA). Submission of the NDA is the first step toward getting approval . . .

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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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