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Watch: Duchenne Newborn Screening – Next Steps Following ACHDNC Dissolution (Webinar Recording)
Last week, PPMD and the Muscular Dystrophy Association provided insights into the current status of the Duchenne nomination to the federal Recommended Uniform Screening Panel (RUSP) for newborn screening following the dissolution of the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC). This briefing provides a timely overview of where the nomination stands,…
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Sarepta: Gene therapy Elevidys has given ‘hope’ to DMD community
It’s been nearly two years since Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for Duchenne muscular dystrophy (DMD) developed by Sarepta Therapeutics, was approved by the U.S. Food and Drug Administration (FDA), marking the first time a gene therapy for DMD was authorized in the U.S. Since its launch, the response to Elevidys has been “overwhelmingly…
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Clinical Research Alert: Observational Study in Female Carriers of SMA and Their Biological Children
Researchers at Natera are seeking female carriers of spinal muscular atrophy (SMA) and their affected or unaffected biological children for an observational study (DYADS study). This study will collect blood samples and health information from participating pairs (mother and child). Findings from this study could help in development of non-invasive prenatal screening tools for SMA.…
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Highlighting a brief history of muscular dystrophy advocacy
I recently penned a column that took a surface look at the history of the disability movement in the United States. As many readers pointed out, my list of world changers and their accomplishments was far from exhaustive. I’d like to share another small part of the movement that has been particularly significant to the…
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BOLLYWOOD BLINGO
Download PDF HERE CHAMPIONS SPONSORSHIP OPPORTUNITIES 2025 The post BOLLYWOOD BLINGO appeared first on CureDuchenne.
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CureDuchenne Raises More than $1M at Napa in Miami to Help Find a Cure for Duchenne Muscular Dystrophy
Premier Wine Tasting and Auction Brought Acclaimed Napa Valley Vintners Together with Miami Philanthropists to Benefit Global Nonprofit CureDuchenne MIAMI, FL, April 16, 2025 – CureDuchenne, a global nonprofit committed to finding a cure for Duchenne muscular dystrophy, and Event Chair Susan Finazzo hosted the fourth annual “Napa in Miami” Wine Tasting and Auction on April 5,…
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MDA Ambassador Guest Blog: How Genetic Testing Helped Us Feel Empowered
Jessica and Mark Lennox live in Jupiter, Florida with their two sons, four-year-old William, and two-year-old old Paul. Mark is a US Army veteran and works in renewable energy. Jess, originally from Maryland, is a full-time Mom. At 3 months of age, William was diagnosed with spinal muscular atrophy with lower extremity predominance (SMA-LED2). Will…
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FDA OKs trial of SRP-9005 gene therapy for LGMD type 2C
The U.S. Food and Drug Administration (FDA) has cleared Sarepta Therapeutics to start dosing and screening in a first clinical trial testing the gene therapy SRP-9005 in people with limb-girdle muscular dystrophy type 2C (LGMD2C). Sarepta said advancement continues for SRP-9003 and SRP-9004, two gene therapies already in clinical testing for other types of LGMD.…
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Quest Podcast: Defending Medicaid with the MDA Advocacy Team
In this Quest Podcast episode, we chat with Joel Cartner, MDA’s Director of Access Policy, and Jori Houck, MDA’s Manager of Advocacy Engagement. They join us to share the most recent updates and information about Medicaid benefits, current legislative efforts and what MDA’s Advocacy Team is doing to protect those efforts, and how you can…
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A primary care visit puts me back on the medical merry-go-round
I have friends and family who seem to be living on a medical merry-go-round. Every day they go to doctor’s appointments, have medical tests, or go through therapy. Their lives revolve around their healthcare. I haven’t been on this type of merry-go-round for a long time. I had a devastating car crash with a drunk…
