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Sarepta Therapeutics Reports Positive Data from Part B of MOMENTUM Study of SRP-5051
Sarepta Therapeutics, Inc. today announced positive data from Part B of SRP-5051-201, the MOMENTUM study.
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PPMD 2023 Duchenne Early Care Meeting Report Published: Enhancing Support in Early Diagnosis
Last year, PPMD hosted a Duchenne Early Care meeting as part of the 2023 Duchenne
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Duchenne Added to Minnesota’s Newborn Screening Panel
PPMD is excited to announce another significant milestone: Minnesota has officially approved the addition of
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Sometimes living with Duchenne is simply too hard
I love to read, but finding time to sit down with a good book can
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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
