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Santhera Receives Approval for AGAMREE® (Vamorolone) as a Treatment for Duchenne Muscular Dystrophy in the United Kingdom
AGAMREE (Vamorolone), whose development at ReveraGen Biopharma was supported by CureDuchenne, has been approved in […]
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Introducing PPMD’s Gene Therapy Hub
We are excited to announce the launch of PPMD’s Gene Therapy Hub! Gene therapy has […]
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Join us at PPMD’s 30th year meetings and events
Happy New Year! As PPMD celebrates its 30th anniversary in 2024, we’re thrilled to announce […]
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Juvena’s JUV-161 named FDA orphan drug for DM1
The U.S. Food and Drug Administration (FDA) has given orphan drug designation to JUV-161, Juvena Therapeutics’ lead […]
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PTC Therapeutics – EMFLAZA® LANDSCAPE IN 2024
Join PTC Therapeutics and CureDuchenne for this prerecorded webinar to hear important information regarding Emflaza, PTC Cares and […]
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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, […]
