January 2025

In August 1985, I was diagnosed with Becker muscular dystrophy. At that time, there was no genetic test for the disease, nor DNA sequencing of the blood. Instead, I underwent a muscle biopsy on my left thigh and calf, the tissue was sent for pathology, and the medical professionals told my parents that while I…

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Dyne Therapeutics, which received early funding from CureDuchenne, plans to pursue US Accelerated Approval from the FDA in 2026 for DYNE-251, an exon-skipping experimental therapeutic for individuals with Duchenne amenable to skipping Exon 51.  Data from their ongoing DELIVER trial, expected Q4 2025, will support this submission, and Dyne is currently enrolling participants for an…

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The Christmas decorations are down and tucked into storage for another year. The house is clean, the counters are clear of the holiday sweets, and our routine is again in sync. However, this holiday season has left lingering fears and sadness quivering in my heart. I share seven children with my husband, Jason: Lexi, 23,…

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In our rapidly evolving therapeutic landscape, there is a critical need for cohesive infrastructure that helps track what happens when individuals are on more than one therapy and how we can continue to evolve standards of care in this new era. This is why PPMD is proud to introduce and begin development of the Dystrophinopathy…

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Avidity Biosciences, Inc. has announced its plans for filing a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for delpacibart zotadirsen (del-zota) for treatment of individuals living with Duchenne muscular dystrophy mutations amenable to exon 44 skipping. Del-zota (AOC 1044) is an exon skipping therapy conjugated to a monoclonal antibody targeting…

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A gene-editing therapy designed to correct a defect in the DYSF gene — one that’s associated with a form of limb-girdle muscular dystrophy, or LGMD — was found to restore dysferlin protein levels and help regenerate muscle tissue in a mouse model of the disease. The researchers are now seeking funding to test the gene-editing approach…

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PPMD is celebrating the 20th anniversary of our endurance fundraising program, Race to End Duchenne, this weekend in Orlando, Florida. 185 team members will participate in the 2025 Walt Disney World® Marathon Weekend, presented by State Farm, running in honor of loved ones affected by Duchenne. The Walt Disney World® Marathon Weekend is a special…

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A few days ago, I watched the Netflix documentary “The Remarkable Life of Ibelin.” I wasn’t prepared for how deeply it would move me. The film follows Mats Steen, a young man from Norway who lived with Duchenne muscular dystrophy (DMD). Online, he was known by the avatar Ibelin, a hero in the game World…

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Capricor Therapeutics has completed its submission of a biologics license application (BLA) seeking U.S. approval of deramiocel, the company’s cell therapy for heart muscle disease in people with Duchenne muscular dystrophy (DMD). “The submission of the BLA marks a pivotal step for Capricor and those impacted by DMD,” Linda Marbán, PhD, CEO of Capricor, said…

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As we enter PPMD’s fourth decade and a new era in Duchenne research, I am once again in awe of this community’s unwavering determination to push boundaries and drive progress. Your generosity continues to propel our initiatives forward in the fight to end Duchenne.  More individuals living with Duchenne have access to therapies than ever…

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