April 2024

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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Dealing with a fracture is a frightening experience for anyone, especially someone living with Duchenne muscular dystrophy, who has weaker bones and is more prone to falling.   Often, you will have to work with emergency room staff who aren’t as familiar with Duchenne and don’t know the signs of fatty embolism syndrome (FES). Then you,…

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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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I had an incident about a month ago that I’ve been thinking about ever since. It’s left me wondering if it’s time for me to begin using a walker, something I tried years ago without much success. The Sunday after my wife, Wendy, had surgery recently, a friend brought me home from church. He dropped…

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Up to eight years of Exondys 51 treatment (eteplirsen or AVI-4658) extended survival in Duchenne muscular dystrophy (DMD) patients over a wide range of ages, a long-term study concluded. Patients treated for longer periods had the lowest risk of death. The study, “Survival among patients receiving eteplirsen for up to 8 years for the treatment…

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Over 60 leading experts in genetics, pediatrics, neurology, and public health advocates have authored a consensus statement in support of implementing Duchenne newborn screening. Key points of the consensus statement include: Timely Intervention: Early screening allows for prompt intervention and therapeutic strategies, slowing disease progression. Reduced Diagnostic Delays: Screening eliminates diagnostic delays, reducing avoidable costs…

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