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REGENXBIO AFFINITY DUCHENNE: Trial of Investigational RGX-202 Gene Therapy
In this webinar, Dr. Naz Dastgir, Executive Medical Director and Clinical Development Lead for RGX-202 […]
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Back to School Resources for Duchenne in 2025
Back to School with Duchenne: Navigating this new 2025 school year is a challenge for […]
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Solid Biosciences Shares Update on INSPIRE DUCHENNE Trial of SGT-003
Solid Biosciences has shared an update on the company’s Phase 1/2 INSPIRE DUCHENNE trial evaluating […]
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Celebrating a long-in-the-works family music songfest
I’m a musician, and so are my brothers, Ron and Tim. We’ve all played professionally, […]
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Calling All Adults with Duchenne & Becker: Join the 2026 PPMD Adult Advisory Committee (PAAC)
Have you been looking for a way to engage with the community more? Do you […]
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MDA Guest Ambassador: Ten Tips for High School Life with a Disability
Madison is from Baton Rouge, Louisiana, and lives with congenital muscular dystrophy. She is an […]
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Solid Biosciences announced a widely anticipated update on their INSPIRE DUCHENNE Phase 1/2 clinical trial of SGT-003
Solid Biosciences announced a widely anticipated update on their INSPIRE DUCHENNE Phase 1/2 clinical trial […]
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Hidden role of MYOD protein may lead to new MD therapies: Study
A protein called MYOD, which is known to control the growth of muscle stem cells […]
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On the importance of human connection
There is power in connection. As humans, we all learn this truth at a young […]
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Update to the U.S. Duchenne Community on the Ataluren (Translarna) for Nonsense Mutation Duchenne Muscular Dystrophy
The FDA is continuing its review of Ataluren (Translarna), the first potential therapy specifically for […]
