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Fin de l’essai clinique : ataluren
Fin de l’essai clinique de l’ataluren pour la dystrophie musculaire Duchenne : dix ans d’espoir
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The holiday season makes me regret not making my kids do chores
The holiday season is upon us, and I am already exhausted. I’m well aware of
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A Thanksgiving Message from Pat
This Thanksgiving, I find myself reflecting deeply on gratitude, especially for this incredible community that
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Watch the Recording: Avidity Biosciences – EXPLORE44-OLE® Topline and Functional Data Presentation
Avidity Biosciences joined PPMD for a community webinar on November 19, 2025, during which the
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Sarepta announces new clinical trial cohort to evaluate enhanced immunosuppression protocol to accompany Elevidys gene therapy in non-ambulant individuals
Sarepta Therapeutics has announced that the FDA has approved Cohort 8 of the ENDEAVOR study,
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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
