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I love my house and the home life my husband, Jason, and I have created. My family lives in a four-bedroom, two-bath, finished-basement, ranch-style home in south-central Nebraska. It was brand-new when we bought it, and we were its first owners. Jason and I had been living in a small rental house with our daughter…

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Leslie Krongold is 63 years old and lives in Mendocino, California where she keeps busy with several volunteer and DIY activities including peer counseling, facilitating online support groups, and organizing accessible walk and roll activities. She was diagnosed at 36 years old with myotonic dystrophy type 1 and has been using a walker at home and…

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In boys with Duchenne muscular dystrophy (DMD), executive skills — essentially the brain’s self-management tools, such as self-control, emotional regulation, and working memory — appear largely typical at age 5, begin to lag around age 8, and show signs of partial catch-up by early adolescence. Those are the findings of a new Swedish study that…

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New research suggests statins may protect adults with type 2 diabetes regardless of how low their predicted heart risk appears. In a large UK study, statin use was linked to fewer deaths and major cardiac events across all risk levels. Even those labeled “low risk” benefited, challenging long-held assumptions about who should receive preventive therapy.…

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Researchers at Biogen are working to better understand spinal muscular atrophy (SMA) and to evaluate a potentially new treatment for babies before they develop any symptoms. The study Newborns with SMA may be eligible to participate in a phase 3 clinical trial (STELLAR-1) to evaluate the safety and efficacy of the investigational therapy salanersen to treat…

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REGENXBIO announced new, positive 18-month functional data from patients treated with the pivotal dose in the Phase I/II portion of the AFFINITY DUCHENNE® trial. They expect to share pivotal topline data in early Q2 2026 and submit a Biologics License Application (BLA) under the accelerated approval pathway in mid-2026.  Link to press release: https://www.prnewswire.com/news-releases/regenxbio-highlights-key-2026-catalysts-and-announces-positive-long-term-functional-outcomes-in-lead-duchenne-gene-therapy-program-302657988.html The post REGENXBIO…

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A new discovery may explain why so many people abandon cholesterol-lowering statins because of muscle pain and weakness. Researchers found that certain statins can latch onto a key muscle protein and trigger a tiny but harmful calcium leak inside muscle cells. That leak may weaken muscles directly or activate processes that slowly break them down,…

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In the book “Good Enough: 40ish Devotionals for a Life of Imperfection,” authors Kate Bowler and Jessica Richie write, “Blessed are you who are buried. You who feel stuck in the depths of grief and despair or who sit in the pit of unknowing.” Since reading this devotional, I’ve been thinking about the phrase “the…

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The Muscular Dystrophy Association (MDA) has opened applications for its 2026 college scholarship program, which provides financial support for students living with muscular dystrophy or a related neuromuscular disease. With the program now in its third year, the scholarships are available to students who are pursuing or planning to pursue full-time undergraduate degrees at accredited U.S.…

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PPMD celebrated a powerful and inspiring Walt Disney World® Marathon Weekend as part of its Race to End Duchenne endurance fundraising program this past weekend! PPMD runners, families, and supporters came together in Orlando, Florida, to raise more than $300,000 to accelerate research, improve care, and strengthen advocacy efforts for the Duchenne and Becker community.…

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