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Pittsburgh, where I now live, receives far less annual snowfall than Shanksville, Pennsylvania, where my wife, Wendy, and I lived for 41 years. Shanksville, in Somerset County, sits squarely in the state’s snow belt. However, every so often, Pittsburgh experiences true winter weather, with bitter cold and significant snowfall. Just a couple of weeks ago,…

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In January, nine states led by Texas revised their challenge to Section 504 of the Rehabilitation Act and the integration mandate required by both Section 504 and the Americans with Disabilities Act (ADA) by arguing in an amended complaint that the mandate, which requires states to fund services in the most integrated setting, is unconstitutional. The Rehabilitation Act, including Section 504, is a landmark…

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We applaud a major legislative victory for the rare disease and Duchenne and Becker community — one made possible because advocates like you raised your voices, shared your stories, and sustained pressure on lawmakers. With the signing of the FY 2026 Consolidated Appropriations Act (H.R. 7148) on February 3, 2026, Congress enacted a powerful package…

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Precision BioSciences announced that the U.S. FDA has cleared its Investigational New Drug (IND) application for a gene editing therapy for Duchenne muscular dystrophy. This clearance allows the company to move forward with a Phase 1/2 clinical study to evaluate safety and early signals of effectiveness.  The investigational therapy uses Precision’s ARCUS® gene editing platform and…

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A Phase 3 trial testing SGT-003, a gene therapy candidate for Duchenne muscular dystrophy (DMD), will start dosing before the end of March, according to Solid Biosciences. The trial follows a successful meeting with the U.S. Food and Drug Administration (FDA), during which the company and the agency aligned on the study’s design. The IMPACT DUCHENNE…

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CureDuchenne Invests in Satellos to Advance a Mutation-Independent Duchenne TherapyLink to press release CureDuchenne is pleased to announce that it has participated in the Satellos Bioscience public offering, supporting the company’s $57.2 million financing to advance its Duchenne muscular dystrophy program through clinical development. The funds will support ongoing and planned clinical trials of SAT-3247,…

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International Women and Girls in Science Day, February 11, endeavors to acknowledge and celebrate the invaluable role that women and girls play in accelerating change and discovery in the professional realm of science, technology, engineering, and math (STEM). In recognition of International Day of Women and Girls in Science, the Muscular Dystrophy Association (MDA) is…

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We all have bad days, but hopefully they are few and far between. Bad days can overwhelm, dampen your spirit, and leave you feeling angry, sad, or depressed. You may need to take a break from whatever has caused them. First, let me clarify that I am talking about bumps in the road, not trauma.…

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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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When the germs of cold and flu season find their way into the Vertin household, they crash our party of nine, and no one is spared. A cold or flu can be especially dangerous for my three sons with Duchenne muscular dystrophy (DMD) — Max, Rowen, and Charlie — because they have compromised immune systems…

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