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Rainy days, Mondays, and FSHD make feeling grateful a challenge
It’s a wintery Monday morning as I write this. My wife, Wendy, left a while
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Wave clinical trial for Duchenne amenable to skipping exon 53 currently enrolling in US
Wave Life Sciences has announced that they are initiating Part C of the FORWARD-53 trial—an
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Simply Stated: Updates in Primary Mitochondrial Diseases
Mitochondria, often called the “powerhouses of the cell,” generate much of the energy that our
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The body trait that helps keep your brain young
Scientists discovered that more muscle and less hidden abdominal fat are linked to a younger
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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
