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DYNE-251 leads to improvements in motor function in DMD: Update
Up to a year of treatment with DYNE-251, Dyne Therapeutics’ investigational exon 51-skipping therapy, led to improvements in motor function for boys with Duchenne muscular dystrophy (DMD), according to a clinical trial update. Based on these positive data from the Phase 1/2 DELIVER clinical trial (NCT05524883), Dyne is launching registrational groups of trial participants, the…
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Raising Our Voices for Duchenne: Recap of PPMD’s Advocacy Webinar
On Thursday, September 5, 2024, PPMD hosted a special webinar, “PPMD Advocacy: Raising Our Voices in 2024 and Beyond,” bringing together advocates to reflect on PPMD’s advocacy efforts in our 30th anniversary year and discuss how we continue to drive policy change for those living with Duchenne and Becker muscular dystrophy. Moderated by PPMD’s Senior…
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Entrada Therapeutics Announces Recipients of Second Annual Diversity, Representation, Equity and Advocacy MatterS (DREAMS) Grant Program
SEE THE FULL PRESS RELEASE BELOW AND HERE – Second annual DREAMS Grant Program awards $25,000 each to three U.S.-based non-profit organizations working to achieve greater equality for those living with Duchenne – – Announced in celebration of World Duchenne Awareness Day, grants are designed to fund efforts advancing diversity, equity, inclusion and accessibility within…
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Myology Course Educates and Advances Next Generation of Neuromuscular Physicians and Scientists
PPMD is dedicated to fighting for every future as we strive to end Duchenne. Since 2011, PPMD has been a proud supporter of the Myology Course, presented by Nationwide Children’s Hospital (NCH) and The Ohio State University which aims to educate and advance the next generation of neuromuscular physicians and scientists. This commitment is essential…
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CureDuchenne Submits Comments on FDA’s Draft Guidance for Industry on Platform Technology Designation Program
CureDuchenne welcomes the recent initiative the Food and Drug Administration (FDA) has taken to improve efficiencies in drug development, manufacturing, and the review process for new drug applications that incorporate designated platform technologies. This initiative is designed to help streamline the drug development and approval process, allowing drug companies to leverage prior knowledge from their…
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Noninvasive qMRI seen to detect muscle changes in LGMD type R1
A noninvasive quantitative MRI, or qMRI, was found to detect early muscle abnormalities among people with limb-girdle muscular dystrophy type R1 (LGMDR1), according to a small study from Europe. Many of the qMRI findings correlated with clinical assessments of muscle function and patient-reported activities. “Our findings revealed alterations in both clinical outcome indicators and qMRI…
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Introducing PPMD’s Newborn Screening Action Center: A Hub for Advocacy and Education
We are thrilled to announce the launch of PPMD’s Newborn Screening Action Center, a powerful resource designed to both educate and empower you as we work to ensure that every baby, in every state, has equal access to timely diagnosis and care. Newborn screening is critical in preventing the delays that can leave families without…
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Dyne Therapeutics Announces Positive New Clinical Data from Phase 1/2 DELIVER Trial of DYNE-251 in Duchenne
Dyne Therapeutics, Inc. has announced positive new clinical data from its ongoing Phase 1/2 DELIVER trial of DYNE-251 in patients with Duchenne who are amenable to exon 51 skipping. DYNE-251 is an exon skipping product that combines a PMO to enable skipping of exon 51 with a fragment antibody (Fab) to increase targeted delivery of…
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Dyne Therapeutics Announces New Clinical Data from Phase 1/2 DELIVER Trial of DYNE-251 in Duchenne Muscular Dystrophy Demonstrating Unprecedented Dystrophin Expression and Functional Improvement in Multiple Cohorts
As an early investor in Dyne Therapeutics, CureDuchenne is pleased share that Dyne announced positive data from their Phase 1/2 trial of DYNE-251 in individuals with Duchenne amenable to skipping exon 51. Individuals treated with 20 mg/kg once every 4 weeks for 6 months showed a mean dystrophin expression of 3.7% of normal when unadjusted…
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Raising Our Voices This Duchenne Action Month
PPMD is excited to join forces with fellow Duchenne-specific organizations from around the world to celebrate World Duchenne Awareness Day on September 7! This global initiative, spearheaded by the World Duchenne Organization, aims to raise awareness about Duchenne and Becker muscular dystrophy and has gained historic significance with the United Nations’ official recognition of the…
