An expression of the live unlimited spirit, stories and voices of the MDA community
It’s St. Patrick’s Day. Have you already picked up your corned beef and cabbage? How about your Irish soda bread? What about your most important seasonal accessory: the MDA Shamrock? Don’t fret! You still have time to buy one (or five). And if you’ve already made a donation, encourage your friends and family to join in . . .
March 16 marks Jerry Lewis’ 90th birthday. No one has been a greater champion for MDA or MDA families than the legendary comedian, and his heroic efforts have helped millions of Americans living with muscle-debilitating diseases and made possible progress in research that was once thought unimaginable. Will you join us in wishing him well . . .
Chris Lorson, professor of veterinary pathology, and molecular biology and immunology at the University of Missouri in Columbia, was awarded an MDA research grant totaling $300,000 over three years to optimize an approach currently in clinical trials for spinal muscular atrophy (SMA) by delivering an enhanced form of drug combined with other potential synergistic therapies. . . .
Jeramiha Hometown: Weston, West Virginia Age: 14 Diagnosis: Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness. It is one of nine types of muscular dystrophy. Favorite School Subject/Activities: I love anything to do with sports! Favorite People and/or Pets: I live with my mom Ashley, stepdad Daniel, and siblings Dakota, Jayleigh, and Malik. . . .
Kurt Harzke’s son Jacob was diagnosed with Duchenne Muscular Dystrophy (DMD) when he was three years old. He’s 13 now, a smart, ambitious teen, who suffers from a disease that currently has no cure. Last month, on Rare Disease Day, February 29, Kurt and Jacob spoke to a group from the Muscular Dystrophy Association (MDA) and . . .
The experimental drug eteplirsen, under development by Sarepta Therapeutics to treat Duchenne muscular dystrophy (DMD), is under review by the U.S. Food and Drug Administration (FDA) for market approval. It marks an exciting and historic time for the MDA community and an important milestone for DMD treatments. Before a final decision is rendered, however, an . . .
Marathon Pharmaceuticals announced today that it is expanding patient access to deflazacort, its investigational therapy for Duchenne muscular dystrophy (DMD). Medical centers across the country are now participating in the open-label expanded access program (EAP) called ACCESS DMD to provide deflazacort to qualified U.S. patients at no charge. Deflazacort, a corticosteroid, works as an anti-inflammatory . . .
It’s the second week of March, and you know what that means: it’s full-on Shamrocks season! Retailers across the country are selling MDA’s iconic paper Shamrocks for $1, $5 or more to help families live longer and grow stronger. Have you added this Twibbon to your social media profiles yet? Now’s the time! Help MDA turn social media green this . . .
For Mindy Scheier, the founder of Runway of Dreams, making a difference for the muscular dystrophy community means thinking both long and short term. Since her son Oliver was diagnosed with a rare form of muscular dystrophy, she has channeled her energy into finding a cure. Together with her family, she has helped raise more . . .
Patients, their families and caregivers, researchers and clinicians are invited to gain insight into the testing process for experimental therapies to treat amyotrophic lateral sclerosis (ALS) via a live webcast this month. The ALS Clinical Trials Guidelines 2016 Workshop, an international effort that seeks to renew and update the current ALS Clinical Trials Guidelines, will . . .
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