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Capricor Therapeutics recently joined PPMD for a community webinar to provide an update on their novel cell therapy, deramiocel (formerly CAP-1002), targeting cardiomyopathy in individuals living with Duchenne. The Capricor team discussed their ongoing Biologics License Application (BLA) process with the U.S. Food and Drug Administration (FDA), highlighting the FDA’s acceptance and priority review of…

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The 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference is underway in Dallas, Texas, bringing together leading experts, researchers, and advocates in the neuromuscular disease community. Today, several significant announcements were made by companies developing therapies for Duchenne. These updates include: Avidity Biosciences Announces Positive Topline Del-zota Data in Phase 1/2 EXPLORE44® Trial Avidity…

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For a quarter of a century, MDA’s Wings Over Wall Street has brought the ALS community together in New York City, raising millions of dollars to advance research and improve care. This year marks the 25th anniversary of the annual benefit, an evening dedicated to honoring extraordinary individuals who are driving progress in the fight…

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This May, the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) plans to vote on whether to add Duchenne to the Recommended Uniform Screening Panel (RUSP) for newborn screening. To support a push for evidence about the impact of early diagnosis on outcomes prior to the vote, PPMD recently hosted the Duchenne Early…

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My husband, Jason, and I are parents to seven children: Lexi, 23; Max, 19; Chance, 17; Rowen, 16; Charlie, 14; Mary, 10; and Callie, 3. This week we’re traveling with five of them: Max, Rowen, and Charlie, who are our three sons with Duchenne muscular dystrophy (DMD), and our two youngest daughters. We’re visiting the…

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Today, the Senate passed a Continuing Resolution (CR) that includes a 57% cut to the Congressionally Directed Medical Research Program (CDMRP). While the CR may keep the government running in the short term, it brings with it concerning implications for Duchenne and Becker muscular dystrophy research. The CDMRP, specifically the Peer-Reviewed Medical Research Program (PRMRP),…

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Grace LoPiccolo, 21, of St. Louis, Missouri, was diagnosed with CMT-1A at the age of nine. She currently attends Saint Louis University majoring in Bioethics and Health Studies, and  Catholic studies. She is currently a Junior but ultimately plans on attending law school and pursuing a field in health and disability policy. Additionally, at Saint…

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The National Organization for Rare Disorders (NORD) is seeking participants for its survey-based study Living Rare, which aims to better understand the real-world lived experiences of people in the U.S. with rare diseases. Living Rare, the first large-scale study of its kind in the U.S., seeks to capture the changing unmet needs and challenges faced by…

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When I was 13 and my brother, Tim, was 16, we got skis for Christmas. I’m not sure why Mom and Dad decided to get them for us. Jeannette High School, my alma mater, had a ski club, but Tim and I weren’t members. I don’t recall asking for skis. Perhaps Tim did. However it…

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Increasing socialization, cultivating meaningful relationships and friendships, and limiting isolation or loneliness in an increasingly remote world is a desire – and challenge – for many adults. Individuals living with disabilities sometimes face additional barriers of physical accessibility and social inclusion in their pursuit of a healthy and fulfilling social life. Add to that a…

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