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Callie Hall lives in Orlando Florida and lives with a unique neuromuscular disability. She is passionate about disability rights, bluegrass mandolin, and being outdoors. RIIIIIIIING the school bell signals the end of the class period.  My teacher says, “Alright everyone, we have finished our week of learning about all the Civil Rights Movements.” Chairs shift…

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The U.S. Food and Drug Administration (FDA) is considering whether to conditionally approve zeleciment rostudirsen (z-rostudirsen), an experimental therapy for Duchenne muscular dystrophy (DMD) in patients with mutations that are amenable to exon 51 skipping. Z-rostudirsen developer Dyne Therapeutics announced that the FDA has agreed to review an application seeking accelerated approval of the investigational…

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Today, Dyne Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for zeleciment rostudirsen (z-rostudirsen) for the treatment of individuals living with Duchenne muscular dystrophy who are amenable to exon 51 skipping. The application has been accepted for Priority Review, and the FDA has assigned a…

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CureDuchenne was an early funder of Dyne Therapeutics, and we are pleased to share that the FDA has accepted the BLA (Biologics License Application) for z-rostudirsen for individuals with Duchenne amenable to skipping exon 51.  This submission is for Accelerated Approval based on dystrophin as a surrogate endpoint, and the FDA has granted the application…

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As the summer flies by, my thoughts turn to going back to school — reluctantly. As a mother of many, the end of summer is nothing new. I have seven children with my husband, Jason: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4. Max, Rowen, and Charlie have Duchenne…

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In recent years, researchers have come to understand that a group of enzymes called histone deacetylases (HDACs) becomes overactive and contributes to the progression of Duchenne muscular dystrophy (DMD). While HDACs play a role in normal muscle regeneration and repair, an imbalance in their activity can have the opposite effect, facilitating muscle degeneration and weakness in…

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One of the biggest misconceptions surrounding ventilation is that it signals the final stage of a neuromuscular disease. This isn’t true. Nazmin Shah “Respiratory interventions are intended to improve symptom management, reduce respiratory complications, enhance life, and, in many cases, prolong overall health,” says Nazmin Shah, a respiratory therapist at a teaching institution in San…

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Commendamide, a metabolite produced by beneficial gut bacteria that are depleted in people with Duchenne muscular dystrophy (DMD), protects muscle cells from damage by enhancing antioxidant mechanisms, a study found. “These findings provide new insights into the gut-muscle axis in DMD” and “support further investigation of microbiota-derived metabolites as postbiotic candidates for DMD therapy,” the…

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Zach Fine Zach Fine and his family know firsthand how imperative it is to have an advocate to educate and fight for your needs while receiving medical care. Zach, a twenty-nine-year-old who lives with Duchenne muscular dystrophy (DMD), broke his femur two years ago and experienced severe complications during his emergency hospital stay. Throughout his…

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I’ve been forced to make lifestyle changes continuously because of the progression of my facioscapulohumeral muscular dystrophy (FSHD) and other health issues. But I’ve regained some independence in the past few weeks after losing some capabilities over the past six months. I have a new sit-to-stand cushion that helps me get up from my wheelchair more safely…

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