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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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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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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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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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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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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…

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My life has recently been a whirlwind of activity. Now that the dust is finally settling, I’ve taken advantage of the relative quiet to take inventory of my family. An inventory of my family? What does that even mean? I have a large family. Inventory is how I describe keeping track of them, their well-being,…

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The muscular dystrophy (MD) community is poised to mark National Muscular Dystrophy Awareness Month with events throughout September to call attention to MD and related neuromuscular disorders — and to raise critical funds for the more than 300,000 U.S. families thought to be affected. The annual observance was established in 2019 by the Muscular Dystrophy…

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The U.S. Food and Drug Administration (FDA) has granted orphan drug status to RAG-18, being developed as a potential treatment for both Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). RAG-18 is a small activating RNA (saRNA) therapy from Ractigen Therapeutics that’s designed to counteract the shortage of dystrophin, the hallmark of both disorders. Orphan drug…

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My wife, Wendy, and I recently enjoyed a beach vacation in North Carolina with our family. That included our three children, Nicole, Jill, and Ryan, as well as their partners and our three grandkids, Iva, Julia, and Theo. Hurricane Debby moved out the day we moved in, and Tropical Storm Ernesto didn’t stir up waves…

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