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Phase 3 trial of SRP-9003 gene therapy in LGMD2E starts screening
Sarepta Therapeutics has started screening participants for a Phase 3 clinical trial called EMERGENE that […]
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CureDuchenne Welcomes Patricia Brown as Senior Director of Community Engagement
Seasoned Healthcare Professional Brings Experience in Patient Advocacy and Community Education to Global Rare Disease […]
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CureDuchenne Announces Educational Events for Families and Caregivers of Individuals with Duchenne or Becker Muscular Dystrophy
Upcoming Events Across the Country Provide Latest Treatment Information and Resources for Managing Challenges of […]
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Caregiving for sons with DMD is like being a frog in boiling water
One of my favorite early-summer memories is from when my sons Max and Rowen were […]
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Next-gen gene therapy SGT-003 named FDA orphan drug
SGT-003, a next-generation gene therapy candidate for Duchenne muscular dystrophy (DMD) being developed by Solid Biosciences, […]
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Astellas Releases Results of Discontinued Phase 1b Study of ASP0367
Astellas has shared results of the Phase 1b study of ASP0367 (MA-0211), an oral investigational drug. The […]
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PPMD Hosts 2024 Duchenne Healthcare Professionals Summit
Each January, PPMD brings together experts in the field for our Duchenne Healthcare Professionals Summit […]
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Turning Challenges into Opportunities: A Conversation With Justin Skeesuck From “I’ll Push You”
Justin Skeesuck, a 48-year-old Idahoan featured in the documentary “I’ll Push You,” started losing the […]
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