A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.
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- MDA Awards Honors Those Driving Progress in Neuromuscular Research and Care
- PPMD Partners with Cooperative International Neuromuscular Research Group, Global Sponsors to Advance Expanded Duchenne Natural History Study
- MDA Ambassador Guest Blog: Redefining How I View Myself
- What’s on my wish list for my family’s Duchenne dream home
- DMD gene therapy can repair muscle fibers but fails to halt tissue scarring
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