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Duvyzat recommended for EU approval as DMD treatment
The Committee for Medicinal Products for Human Use, an arm of the European Medicines Agency […]
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Watch: Sarepta Therapeutics – Duchenne Gene Therapy Update (Webinar Recording)
Sarepta Therapeutics recently joined PPMD for a community webinar to share the latest updates on […]
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The BELS Questionnaire: A New Tool to Support Your Child’s Emotional Wellbeing
PPMD is excited to share that a new tool, the BELS (Behavioral, Emotional, Learning, and […]
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Simply Stated: Updates in Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic lateral sclerosis (ALS) is a rare, progressive neurodegenerative disease that leads to muscle weakness, […]
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MDA Ambassador Guest Blog: How the Power of a Few People (and a Dog) Can Change Your World
David Daw is 56 years old and lives in Kingston New York with his wife […]
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Top 5 Things to Know About MDA’s Efforts to Protect Medicaid
In 2025, MDA’s advocacy team has focused on protecting Medicaid from Congressional budget cuts. We […]
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My son’s Make-A-Wish experience at WrestleMania was unforgettable
I’m writing this column as my husband, Jason, drives our family through the last rolling […]
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Why Multidisciplinary Care for ALS Yields Better Outcomes
For those living with neuromuscular diseases, including amyotrophic lateral sclerosis (ALS), access to quality care […]
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CureDuchenne set to host 2025 Futures conference in San Antonio
CureDuchenne will host its annual Futures National Conference May 22-25 in San Antonio, as part […]
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Reflecting on 2 positive years of life, love, and Duchenne
Two days ago I marked the second anniversary of my relationship with my girlfriend, Amanda. […]
