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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
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Avidity’s del-desiran for DM1 named orphan drug in Japan
The Japan Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug status to
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Travelers with Disabilities Share Their Top Tips to Avoid Mishaps
Travel blogger Cory Lee lives with spinal muscular atrophy (SMA) and travels with his 400-pound
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Setback in gene therapy for Duchenne muscular dystrophy as immune system emerges as key barrier
A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short,
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Congress Moves Forward on Medicaid Cuts: What You Need to Know
This week, we received concerning news from Washington: Congress has passed a budget resolution that
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Vamorolone up for approval in Canada as treatment for DMD
Health Canada has agreed to review an application by Kye Pharmaceuticals seeking the approval of
