January 2025

By Debra Miller, Founder and CEO of CureDuchenne  Duchenne International Outreach: Bridging Gaps in Duchenne Care  At CureDuchenne, we have always believed in the transformative power of connection—the kind of connection that goes beyond borders, languages, and cultures to bring care, hope, and possibility to families affected by Duchenne muscular dystrophy. In 2024, through the…

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Once a week treatment with a corticosteroid was seen to lower some biomarkers of muscle damage and inflammation, helping to improve muscle function in people with some forms of muscular dystrophy, according to a pilot study in the U.S. Prednisone was given once weekly for six months to 19 people with limb-girdle muscular dystrophy (LGMD),…

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The Race to End Duchenne program is celebrating our 20th anniversary in 2025, and we spent the weekend of January 8-12 kicking it off at the place where it all began – the Walt Disney World Marathon Weekend, presented by State Farm! It was a special weekend for our community, where we came together to…

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I live in rural Nebraska with my husband and our seven children. Three of them — Max, 19; Rowen, 15; and Charlie, 14 — live with Duchenne muscular dystrophy (DMD). People who meet me in person for the first time when I’m with my family are often curious to know what kind of vehicle we…

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Everyone can help advance the research evolution for Duchenne and Becker. Bringing data sources together from multiple research endeavors is a key component of PPMD’s plan to advance the research evolution, and The Duchenne Registry has the ability to represent all families in this effort. Started in 2007, The Duchenne Registry is PPMD’s patient-driven research…

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The investigational therapy DYNE-101 worked as expected and led to functional improvements for people with myotonic dystrophy type 1 (DM1) in a clinical trial, according to an update from developer Dyne Therapeutics. The Phase 1/2 ACHIEVE trial (NCT05481879) will now enroll a new group of participants who will all receive the dose that’s so far…

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As the 119th Congress and the 47th Administration prepare to take office, PPMD is committed to furthering the needs of our community through comprehensive, creative, and tenacious advocacy on Capitol Hill, regardless of politics. Over the years, we have earned the trust and respect of legislators across the political spectrum to support our mission to…

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PPMD is excited to host the sixth in-person Duchenne Healthcare Professionals Summit in San Diego, California, January 15-17. Since the Summit’s inception in 2018, the event has brought together thousands of healthcare providers, researchers, leaders in federal advocacy, and industry partners for discussion, collaboration, and connection.  Over three full days of meetings, over 350 attendees…

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The U.S. Food and Drug Administration (FDA) has granted IPS Heart’s GIVI-MPC — a stem cell therapy designed to make new muscle tissue in people with muscular dystrophy, or MD — orphan drug status for the treatment of Becker muscular dystrophy (BMD). This designation aims to encourage the development of treatments for rare diseases, or those…

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Many times over the years since the 1989 car crash that nearly killed me, I’ve been asked if I had any near-death or out-of-body experiences while I hung between life and death. I always try to clarify what people mean by these terms. If we’re talking about my spirit hovering in the corner of a…

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