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Accessible Travel on a Budget: Smart Tips for Exploring Affordably
Travel opens doors to new perspectives, cultures, and experiences. But for many, exploring the world can seem like an unattainable luxury — too expensive, too complicated, or simply inaccessible. The good news? Affordable and accessible travel is more possible than ever with the right planning and resources. Whether you’re dreaming of a weekend getaway or…
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CureDuchenne Ventures Invests in Entos Pharmaceuticals for Development of a Redosable, Full-Length Dystrophin Gene Therapy
Newport Beach, Calif. and Edmonton, Canada (May 22, 2025) – CureDuchenne Ventures announced an initial $1M investment in Entos Pharmaceuticals Inc (Entos), a biotech company that develops genetic medicines utilizing its non-viral, redosable Fusogenix PLV delivery platform. Entos will use this funding to create a muscle-targeting therapeutic to deliver full-length dystrophin for the treatment of…
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CureDuchenne Appoints Brenda Wong, MD, as Chief Medical Advisor to Advance Research and Improve Care for Individuals with Duchenne and Becker
Newport Beach, Calif., May 21, 2025 — CureDuchenne, a global nonprofit committed to finding and funding a cure for Duchenne muscular dystrophy, proudly announces the appointment of Brenda Wong, MD, as its new Chief Medical Advisor, effective January 5, 2026. A nationally recognized authority in neuromuscular medicine and clinical research, Dr. Wong has devoted her career…
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Satellos reports data from their Phase 1b study
Satellos Biosciences announced their Phase 1b results from their open-label study with SAT-3247, which aims to improve muscle regeneration in Duchenne. SAT-3247 was safe and well-tolerated in 5 individuals with DMD that were treated for 28 days, and encouraging exploratory functional measurements were observed, prompting plans for follow-up studies. Satellos will be presenting at CureDuchenne’s…
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Satellos Announces Positive Functional Data from Phase 1b Open-Label Trial of SAT-3247 in Adults with Duchenne
Satellos Bioscience Inc. has announced encouraging data from the company’s Phase 1b open-label study of SAT-3247, an oral small molecule inhibitor of AAK, designed to address progressive muscle loss by restoring muscle regeneration in response to damage. The 28-day, early-stage trial included five adult Duchenne patients (ages 20–27), and demonstrated early signs that SAT-3247 may…
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How to Manage Stress and Fear in Uncertain Times
People in the neuromuscular disease community are hearing many different reports about policy and funding changes that could affect their lives or their loved ones. This has many people feeling uncertain. “I see that the community is understandably concerned,” says Mark Fisher, MDA’s Director of Advocacy Engagement. “They’re nervous and worried that programs they rely…
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Investigational gene therapy shows benefits in two boys with DMD
An investigational gene therapy called GNT0004 has demonstrated sustained stabilization of motor function at up to two years of follow-up in two boys with Duchenne muscular dystrophy (DMD). The boys received what was determined to be the effective dose of GNT0004, which was developed by Genethon, during the initial Phase 1/2 of a multiphase clinical trial…
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Florida Signs Duchenne Newborn Screening into Law
We are delighted to share exciting news for the Duchenne community: Governor Ron DeSantis has officially signed Florida’s Duchenne newborn screening legislation (HB 1089 / SB 524) into law. With this historic act, Florida becomes the latest—and one of the largest—states to add Duchenne to its newborn screening panel. This achievement marks a major milestone…
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MDA Conference Grants Support Resources to the Community
The Muscular Dystrophy Association (MDA) provides a significant number of research grants each year to scientists working in the field of neuromuscular diseases. But what many may not know is that MDA also provides grants to other organizations that provide services to individuals living with specific neuromuscular conditions, in the form of conference grants that…
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How my fight for inclusive access to a soccer stadium paid off
As a Duchenne muscular dystrophy survivor, I’ve spent much of my life on the outside looking in, especially when it comes to public events like concerts and sporting events. My condition affects not only my ability to move but also my breathing. I rely on a BiPAP ventilator 24/7, and someone must always be by…
