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CHMP Issues Negative Opinion for Renewal of Conditional Marketing Authorization for Translarna™ (ataluren) in Europe
PPMD is disappointed to learn that the European Medicines Agency (EMA)’s Committee for Medicinal Products
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Coping with seasonal affective disorder and FSHD as best I can
The symptoms of my facioscapulohumeral muscular dystrophy (FSHD) make staying positive a daily challenge. Many
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PepGen Announces First Patient Dosed in CONNECT1-EDO51 Phase 2 Clinical Trial of PGN-EDO51 for Duchenne Muscular Dystrophy Patients Amenable to Exon 51 Skipping
As an early funder of PepGen, we are pleased to share that PenGen has dosed
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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
