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 “Disco For Duchenne” Gala Featured Dancing, Drinks, a Culinary Experience and More  to Help Advance Research for the Rare Disease  AUSTIN, Texas., April 16, 2024 – CureDuchenne, a leading global nonprofit focused on finding and funding a cure for Duchenne muscular dystrophy, and the Revell family of Austin hosted the 15th annual fundraiser gala, Champions to…

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Treatment with an antioxidant supplement led to improvements in muscle strength and quality of life for people with facioscapulohumeral muscular dystrophy (FSHD) in a small clinical trial. The use of such supplements was particularly seen to improve the muscle quality of patients’ quadriceps — a group of muscles found on the front of the thigh.…

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Edgewise Therapeutics, Inc., has announced positive two-year topline results from the ARCH open label study of sevasemten (EDG-5506) in adults with Becker. Sevasemten is an orally administered small molecule inhibitor designed to prevent contraction-induced muscle damage in dystrophinopathies, including Becker and Duchenne. Results showed that during two years of sevasemten treatment, participants’ North Star Ambulatory…

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If you have a desire to drive with Duchenne muscular dystrophy or be a passenger in a wheelchair-accessible vehicle, there are plenty of opportunities to make that dream a reality. This blog post can help guide you through the complexities of finding funding for an accessible vehicle and adaptive driving aids, evaluating the needs you…

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Edgewise Therapeutics Announces Positive Two-Year Topline Results from the ARCH Open Label Trial of Sevasemten (EDG-5506) in Adults with Becker Muscular Dystrophy (Becker) – The North Star Ambulatory Assessment (NSAA) remained stable relative to declines reported in Becker natural history studies – – Significant decreases were observed in circulating levels of creatine kinase (CK) and…

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PPMD is proud to announce the launch of our first state-specific Duchenne Advocacy Day in Illinois! This initiative marks a significant milestone in PPMD’s efforts to advance legislative progress for Duchenne at the state level. PPMD has long been at the forefront of Duchenne advocacy efforts. Its annual Advocacy Conference stands as the longest-running advocacy…

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For more than a decade, PPMD has been at the forefront of advocating for Duchenne newborn screening. In 2023, we expanded our ongoing work in the space by conducting a parent survey to build evidence and understanding of the benefits of newborn screening for Duchenne. The results of the study have been published in the…

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Diagnostic testing of individuals in India suspected of having limb-girdle muscular dystrophy (LGMD) revealed two novel mutations in the calpain-3 gene, known as CAPN3, that were found to cause the common subtype R1, known as LGMDR1. These findings were detailed in a new study reporting the results of testing in more than 30 people who…

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Southern California’s Premier Wine Event Brings Together Acclaimed Napa Valley Vintners   Under One Roof to Raise Funds for CureDuchenne    NEWPORT BEACH Calif., March 8, 2024 – Marking a significant milestone, Napa in Newport benefiting CureDuchenne will commemorate its 10th anniversary on April 20, 2024, at the luxurious Pendry Newport Beach. Esteemed for its unparalleled selection…

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I have three sons with Duchenne muscular dystrophy (DMD): Max, 18, Rowen, 15, and Charlie, 13. When Max was young, he met all of his developmental milestones late. He didn’t sit up until he was 11 months old, crawl until after his first birthday, or walk until he was 17 months old. I imagined every…

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