Seeing Both Sides of the Plastic Straw Controversy
A few years ago, if you had asked me what it means to be eco-friendly, I would have given you the average answer: “Turning ... Read more
Updates
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Jiffy Lube, MDA Join Forces for 7th Annual MUSCLE UP! Campaign
Jiffy Lube is teaming up with the Muscular Dystrophy Association (MDA) in the annual MUSCLE UP! campaign for the seventh year in a row. The campaign, which ... Read more -
2 Nonprofits Join Forces to Help Duchenne Patients Fight Insurance Denials
Two small patient advocacy groups, Little Hercules Foundation and Team Joseph, are stepping up their joint Duchenne Family Assistance Program (DFAP). The expanded program, created ... Read more -
First Patient Enrolled in Phase 2 Trial of MNK-1411, Mallinckrodt’s Investigational Therapy for DMD
The first patient has been enrolled in a Phase 2 trial evaluating the effectiveness and safety of MNK-1411, an investigational therapy for Duchenne ... Read more -
Arizona Woman with Duchenne is a Rarity Among the Rare
Duchenne muscular dystrophy affects an estimated 1 in 3,500 male births. But Elizabeth Heller is an outlier even among her rare friends. The Chicago-born ... Read more
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How Lyrica Has Affected My Relationship with Food
All my life, I’ve been very lucky not to be burdened with food and ... -
$700,000 MDA Grant to Help Establish LGMD Clinical Research Network
Nicholas Johnson, vice chair of research and associate professor of neurology at Virginia Commonwealth ... -
New Online Resource, DuchenneXchange, and Its Offerings Explained in Aug. 7 Webinar
The nonprofit group CureDuchenne and Connecticut-based rareLife Solutions have launched DuchenneXchange — a dedicated online platform where those ... -
Family’s Quest for ‘Overlooked’ Duchenne Treatments Leads to Breast Cancer Medicine Tamoxifen
Gavin Ward, a spunky 8-year-old Arkansas boy with Duchenne, enjoys building contraptions with his ... -
FDA Places Hold on Phase 1/2 Trial of Sarepta’s Gene Therapy for DMD
A Phase 1/2 trial evaluating Sarepta’s microdystrophin gene therapy candidate for Duchenne muscular dystrophy (DMD) was ... -
Consortium.AI to Advance New Treatments for Duchenne Using Artificial Intelligence
Insilico Medicine and A2A Pharmaceuticals, two leading artificial intelligence (AI) biotechs, are collaborating to ...
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FDA Grants Rare Pediatric Disease Status to Myonexus’ MYO-101 for LGMD 2E
News
The U.S. Food and Drug Administration recently granted rare pediatric disease designation to Myonexus Therapeutics’ experimental gene therapy MYO-101 for the treatment of limb girdle muscular dystrophy (LGMD) type 2E, or LGMD2E. The announcement was made by Myonexus and Nationwide Children’s ... Read more