December 2025

Katie Brooks is a mom of two incredible little boys, Dominic and Daniel. She was born and raised in Austin, TX and moved to Atchison, KS to attend college at Benedictine College. She has worked in special education since 2007 and is currently an Early Childhood Special Education Coach for the Shawnee Mission School District in…

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Clinical trials are the cornerstone of research. When a new medical treatment, device, or strategy is being developed, researchers need to know how it will perform in humans. Will it be helpful, harmful, or no different from the available alternatives? Investigators try to answer these questions through clinical trials. “A clinical trial is an experiment…

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Duchenne muscular dystrophy (DMD) causes dysfunction of muscle cells that help move blood through the circulatory system, a study found. The changes associated with DMD included dysregulated activity of some genes and altered dynamics and structure of mitochondria, the cell’s powerhouses. “These findings highlight the importance of targeting vascular [blood vessel] abnormalities in therapeutic strategies…

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A recent 60 Minutes segment examining the cost of gene therapy put a national spotlight on a question families living with rare disease confront every day: how can lifesaving medical breakthroughs exist if they remain out of reach for the people who need them? The public reaction to the multimillion-dollar price tags for gene therapies…

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Stepping into this role in such a historic year has been humbling and inspiring. This is my first End of Year message as MDA’s President and CEO, and I want to begin by thanking you. Our progress in 2025 was driven by the partnership we share across this entire community of families, clinicians, researchers, volunteers,…

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The idiopathic inflammatory myopathies (IIMs) are a group of rare autoimmune muscle diseases that include dermatomyositis, polymyositis, immune-mediated necrotizing myopathy (IMNM), antisynthetase syndrome, and inclusion body myositis (IBM). These disorders cause progressive muscle weakness, can affect multiple organs, and often impact quality of life. Together, the IIMs affect an estimated 2 to 25 per 100,000…

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I’m feeling funny today — humorous, in fact. Brimming with sarcasm and a splash of laughter, today feels like a cocktail, served over ice and garnished with a tiny umbrella and an extra-long, twisty straw. The main ingredient? Wit, with a dash of mischief. Why? Why not. Every so often, I revisit my past columns…

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PPMD recently held a community webinar with Capricor Therapeutics, during which the company shared their most recent results from the Phase 3 HOPE-3 trial evaluating deramiocel (CAP-1002), Capricor’s investigational cell therapy for the treatment of Duchenne muscular dystrophy. The presentation included a review of the data, a discussion of planned next steps for FDA engagement…

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CureDuchenne applauds the U.S. Department of Health and Human Services (HHS) for adding Duchenne muscular dystrophy to the Recommended Uniform Screening Panel (RUSP) for newborn screening. We also extend our congratulations to Parent Project Muscular Dystrophy (PPMD) for leading the nomination and to Muscular Dystrophy Association (MDA), the co-sponsor. Several pilot newborn screening programs—including one supported by CureDuchenne—were instrumental in…

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A presentation with Dr. Doug Kerr, Chief Medical Officer at Dyne Therapeutics. Dr. Kerr shares topline results from the DELIVER clinical trial, along with new long-term data showing sustained benefits out to 24 months. DELIVER is Dyne’s global, randomized, placebo-controlled, double-blind, Phase 1/2 trial that evaluated the safety, tolerability and efficacy of zeleciment rostudirsen (z-rostudirsen,…

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