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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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, […]
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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 […]
