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CureDuchenne Webinar with Dyne Therapeutics | Advancing the Promise of FORCE to Deliver for Patients
Dyne Therapeutics will present initial clinical data, first released on January 3rd, 2024, from the […]
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15th Annual Champions to CureDuchenne Returns to The University of Texas Golf Club on April 6 to Find a Cure for Duchenne Muscular Dystrophy
“Disco For Duchenne” Gala Features Dancing, Drinks, a Culinary Experience and More to Help Advance […]
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New cell-based study reveals autoimmune mechanism in DM2
A cell-based study revealed the biological mechanism behind the increased tendency for people with myotonic […]
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Breaking news: Rare Disease Day Congressional Hearing to Address the BENEFIT Act
On Thursday, February 29th, Rare Disease Day, the House Energy and Commerce Committee will hold […]
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In choosing a college, my son with DMD is already winning
I am a competitive former college athlete married to a someone who is equally competitive […]
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Revised Community-Led Duchenne Guidance Published: Developing Potential Treatments for the Entire Spectrum of Disease
Today PPMD is pleased to share that the revised community-led Duchenne Guidance has been published […]
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Exon-skipping AOC 1044 gets FDA rare pediatric disease status
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to AOC […]
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Watch: Overview of WVE-N531 and the FORWARD-53 Clinical Trial with Wave Life Sciences (Webinar Recording)
Wave Life Sciences recently joined PPMD for a community webinar to share an update on […]
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SAT-3247 improves muscle function in FSHD mouse model
SAT-3247, an oral therapy candidate for rebuilding muscle tissue, improved muscle function in a mouse […]
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I’m tapering off antidepressants and steroids with no regrets
On Feb. 19, I went to see my psychiatrist for the first time in 2024, […]
