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 As an early investor in Dyne Therapeutics, CureDuchenne is happy to share that new analyses out to 24 months showed improvement in heart and lung function with z-rostudirsen compared to declines observed in published Duchenne natural history data.    Link to press release:  https://investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-new-positive-cardiopulmonary-results The post Dyne reports cardiopulmonary benefits in Duchenne trial of z-rostudirsen in…

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Santhera Pharmaceuticals announced results from the ongoing Guardian study in Duchenne, and showed that AGAMREE had comparable effectiveness compared to historical cohorts taking prednisone or deflazacort, based on time to loss of ambulation.  Moreover, individuals taking AGAMREE had fewer vertebra fractures and less incidence of cataracts compared to those taking deflazacort.  Individuals taking AGAMREE also…

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Quest Media is an innovative, adaptive lifestyle platform from MDA. With the power of this platform, we foster awareness and empowerment and have important conversations with experts, thought leaders, and members of the neuromuscular disease community about topics that matter to them and to the larger community of individuals with disabilities. With so many valuable…

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Each year, MDA presents awards to recognize people who are making a lasting impact on research and care for people living with neuromuscular diseases. From scientists to clinicians to advocates, the 2026 recipients reflect the depth of excellence and leadership across the neuromuscular field. Meet this year’s honorees. 2026 MDA Legacy Award for Achievement in…

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PPMD is proud to share our partnership with the Cooperative International Neuromuscular Research Group (CINRG) to advance the expanded Duchenne Natural History Study (eDNHS) study. Joining PPMD in support of the eDNHS study is Insmed Gene Therapy, LLC; ITF Therapeutics, LLC; NS Pharma, Inc; Santhera Pharmaceuticals (Switzerland), Ltd; Sarepta Therapeutics, Inc; Solid Biosciences Inc; TRiNDS,…

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Stephanie Chicas is 32 years old and lives in Alexandria, VA. Stephanie has SELENON congenital muscular dystrophy and uses a ventilator via a tracheostomy. She loves cuddling with her cat, Matcha, while reading a book and enjoying a cup of matcha latte. Stephanie also enjoys going for walks in the park, attending dance class, and…

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My family needs more space. I wrote about this recently, and we have since decided not to remodel our current home. Since there’s no room to add on, we’re now focused on finding a new, larger house. We have a big family. My husband, Jason, and I share seven children: Lexi, 24; Max, 20; Chance,…

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A new 3D model of Duchenne muscular dystrophy (DMD) reveals that while gene therapy can bolster muscle strength, it may be unable to halt the progressive scarring that drives the disease, according to a study by researchers at Genethon. Findings suggest that “microdystrophin” gene therapies, similar to several currently in clinical use, successfully improve muscle…

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Researchers at Natera are seeking female carriers of Duchenne/Becker muscular dystrophy (DMD/BMD) and their affected or unaffected biological children for an observational study (DYADS study). This study will collect blood samples and health information from participating pairs (mother and child). Findings from this study could help in development of non-invasive prenatal screening tools for DMD/BMD.…

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On Feb. 19, my girlfriend, Amanda, and I presented at a seminar titled “AI in Social Work” at the National University of Singapore’s School of Computing, on behalf of our accessible art studio, Rebirth Ensemble. It explored how we can harness artificial intelligence (AI) to drive social impact by supporting accessibility innovations for vulnerable populations.…

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