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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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While February may be known for Valentine’s Day candy and flowers, it is also American Heart Month!  Within the dystrophinopathy community, keeping heart health front and center is especially critical. It is estimated that around 67% of mothers of children with Duchenne muscular dystrophy and nearly 90% of mothers of children with Becker muscular dystrophy…

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Napa’s Finest Come to Newport Beach, Led by Vintner Chair Peter Michael Winery  NEWPORT BEACH, California (February 5, 2026) – CureDuchenne, a global leader in accelerating research and care for Duchenne muscular dystrophy, has announced that its annual Napa in Newport event will debut as an elevated weekend-long experience, showcasing the best of Napa Valley in coastal luxury. Taking place March…

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Free nationwide events deliver expert guidance and meaningful connections for families impacted by Duchenne and Becker muscular dystrophy  NEWPORT BEACH, Calif., February 4, 2025 – CureDuchenne, a global leader in advancing research and improving patient care for individuals with Duchenne and Becker muscular dystrophy, today announced the 2026 lineup of CureDuchenne CARES events, designed to deliver the latest treatment insights,…

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