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I welcome Agamree’s approval with renewed optimism
I received an email on Jan. 12 from Duchenne UK, an important Duchenne muscular dystrophy […]
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Keep Dreaming for the Future – Even with DMD
By Jacob Gapko Jacob Gapko is 46 years old and has Duchenne muscular dystrophy. He uses […]
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Sarepta Therapeutics Announces Positive Data for Duchenne Muscular Dystrophy Amenable to Skipping Exon 51
Sarepta Therapeutics reported positive data for SRP-5051, their next-generation exon skipping agent designed for individuals amenable […]
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‘Favorable risk-benefit profile’ seen for DMD treatment SRP-5051: Trial
SRP-5051 is able to increase the production of dystrophin protein in people with Duchenne muscular […]
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Watch: Navigating Steroid Choices – Understanding the Process, Pathways, and Policies (Webinar Recording)
Last week, PPMD was joined by Kathy Mathews, MD (Neurologist), Christina Trout, RN, MSN (Clinic […]
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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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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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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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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 […]
