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Wake up to the achievements of the disability rights movement
This August will mark 40 years since I was diagnosed with what we eventually discovered […]
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The Future of Newborn Screening for Duchenne: Moving Forward After the ACHDNC’s Dissolution
Yesterday, we received news that the Advisory Committee on Heritable Disorders in Newborns and Children […]
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Living with Duchenne muscular dystrophy feels like a gamble
I don’t gamble. I don’t know how to do it. I wouldn’t even know how […]
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Potential DMD treatment KER-065 shows safety in Phase 1 study
KER-065, a potential treatment for Duchenne muscular dystrophy (DMD) and other neuromuscular diseases, showed a […]
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One Family’s Decades of Dedication to MDA Summer Camp
Craig Helget and his brother, Terry Helget, at MDA Summer Camp in 1985. The Helget […]
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Recap: PPMD Together: Minneapolis, MN
This past weekend, PPMD was joined by families, individuals with Duchenne and Becker, clinicians, researchers, […]
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Quest Podcast: Finding Joy in the Midst of Change
In this Quest Podcast episode, we chat with MDA Ambassador Jess Westman about embracing individuality […]
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Watch: Avidity Biosciences – Topline Data from EXPLORE44® Clinical Trial (Webinar Recording)
Avidity Biosciences recently joined PPMD for a community webinar to share topline data from their […]
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MDA 2025: Benefits seen for 3 OPMD patients given gene therapy
The use of experimental gene therapy BB-301 led to improvements in swallowing ability for the […]
