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A little over a year ago, neurologist Sarah Wright administered Elevidys (delandistrogene moxeparvovec-rokl) to then 5-year-old Hiram Secrist, making him the first Duchenne muscular dystrophy (DMD) patient to receive the gene therapy outside of a clinical trial. Elevidys became the first and only gene therapy available for DMD patients after the U.S. Food and Drug…

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My past two columns highlighted significant life events: my daughter’s wedding and my return to the workforce. I’m keeping that theme going today as well, noting my oldest son’s move into his college dorm! Having children leave to experience lives of their own, outside the home you’ve provided for the first 18 years of their…

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PPMD is excited to announce another significant milestone: Massachusetts has officially approved the addition of Duchenne to the state’s newborn screening panel! The addition of Duchenne to the Commonwealth’s newborn screen panel was included as an amendment to the Maternal Health bill (H. 4999) that was signed Friday, August 23, by Governor Healey and will…

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 Dallas’ Finest Will Unite to Advance Innovative Research and Care for Duchenne Muscular Dystrophy  Dallas, TX – August 19, 2024– CureDuchenne, a leading nonprofit dedicated to finding a cure for Duchenne muscular dystrophy, is thrilled to announce its upcoming Champions in Dallas event. Set to take place on Thursday, October 3, 2024, at the stunning…

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The U.S. Food and Drug Administration (FDA) has given WVE-N531, an investigational therapy for Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping, a rare pediatric drug designation. This status aims to incentivize companies developing treatments for serious or life-threatening conditions that primarily affect children and are considered rare, defined in the U.S. as…

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Earlier this month, I attended the debut of Stephanie Esther Fam’s play “Absence” at Singapore’s Gateway Theatre Black Box. Fam, a leading Singaporean poet and playwright in disability-led theater, is also a close friend of mine. Although I had known of her work for some time, we first connected on social media over a poem…

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Dyne Therapeutics recently joined PPMD for a presentation of clinical data from the DELIVER Trial of DYNE-251 in individuals living with Duchenne muscular dystrophy who are amenable to exon 51 skipping, first released on May 20th, 2024. Dyne’s Chief Medical Affairs Officer and VP of Global Patient Advocacy shared insights on how these data demonstrated…

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International-Outreach-and-World-Duchenne-Day-2024-FINALDownload Newport Beach, CA – August 15, 2024 — CureDuchenne, a global leader in research, patient care and innovation for improving and extending the lives of those with Duchenne muscular dystrophy, proudly recognizes World Duchenne Day, celebrated on September 7, by launching new global initiatives and amplifying awareness of the disease. Duchenne muscular dystrophy is…

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I have seven children, and three of them are living with Duchenne muscular dystrophy (DMD): Max, 18, Rowen, 15, and Charlie, 13. If you’re a caregiver or a parent, I’m sure you’ll understand how tired I am. I won’t peruse my past columns, but I’m sure I’ve written about how worn out I get. So…

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Roughly four decades into living with limb-girdle muscular dystrophy, I’ve developed numerous ways to thrive. Like many others, I believe in the power of a positive attitude. I try my best to project a positive outlook when communicating with others, whether it’s face to face or by using words in a column that project out…

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