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PPMD Launches New Study to Understand Patient and Caregiver Treatment Preferences
PPMD has a long history of working alongside our community to document treatment preferences and […]
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A Government Shutdown Couldn’t Stop Advocates During MDA on the Hill 2025
On November 2-4, 2025, despite a historic government shutdown, MDA returned to Washington, D.C. for […]
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Myotonic dystrophy drug SRP-1003 trial advances to higher doses
A Phase 1/2 clinical trial testing SRP-1003, a treatment for myotonic dystrophy type 1 (DM1), […]
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Sarepta Announces FDA Approval to Begin ENDEAVOR Cohort 8 Dosing in Non-Ambulatory Individuals Living with Duchenne
Sarepta Therapeutics, Inc. has shared that the U.S. Food and Drug Administration (FDA) has approved […]
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MDA Ambassador Guest Blog: Breaking the Chains of Stigma in My Arab Community
Samaher (Sam) Abuzahriyeh is 33 years old and lives in Millbrae, CA. She was diagnosed […]
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Immune cells use a surprising trick to heal muscle faster
A research team has found that specific immune cells can connect with muscle fibers in […]
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I’m conflicted about the idea of a cure for muscular dystrophy
I am conflicted about the word “cure.” Dictionary.com defines the noun in several ways, including: […]
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I went from lifting 150 pounds to carrying the weight of Duchenne
I met my husband, Jason, when we were both student athletes at a small college […]
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Clinical Research Alert: Natural History Study of Individuals with Cardiomyopathy Associated with FRDA
Researchers at Lexeo Therapeutics are seeking individuals with cardiomyopathy associated with Friedreich’s Ataxia (FRDA) to participate […]
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Letters from Lily: Advice for Parents of Children with Neuromuscular Disease
Hello! For those of you who don’t already know me, my name is Lily and […]
