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Solid Biosciences, Inc., has shared updates on the company’s gene therapy program for the treatment of Duchenne. According to Solid, 33 participants have now been dosed in the ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial of SGT-003. SGT-003 is a gene therapy candidate for the treatment of Duchenne that delivers a microdystrophin via their novel…

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Avidity Biosciences has released answers to frequently asked questions about their Managed Access Program (MAP) for individuals with Duchenne who are amenable to exon 44 skipping and meet clinical criteria. Learn more about eligibility and access: Avidity MAP FAQ Advocacy Community January 2026Download The post Avidity Biosciences Managed Access Program (MAP) – Frequently Asked Questions…

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Using a “pothole-filling” strategy that combines the precision of DNA-like strands with the versatility of small molecules, scientists have designed a new type of therapy that can target the mutant genetic material that causes myotonic dystrophy type 1 (DM1). This hybrid approach allows scientists to stabilize tangled RNA structures and prevent them from trapping vital…

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In this Quest Podcast episode, we chat with public speaker, consultant, and disability advocate, Jax Cowles. Jax shares an honest, thoughtful, and deeply creative conversation about daily life, independence, and problem-solving.  She opens up about how creativity and “life hacking” became essential tools rather than optional skills, and how small, low-cost adaptations can completely transform…

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Solid Biosciences reports that 33 children have now been dosed in their SGT-003 gene therapy trial for Duchenne. So far, the treatment has been generally well tolerated with signs of improved muscle and heart biomarkers. Their global Phase 3 trial is also underway, with the first participant enrolled. Press Release: https://investors.solidbio.com/news-releases/news-release-details/solid-biosciences-provides-2026-outlook-underscoring Community Letter: https://www.solidbio.com/letter-to-the-duchenne-community-2/ The post Solid Biosciences Reports Progress…

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For people living with neuromuscular diseases, driving can be more than a convenience — it can be a road to independence. Chad Strowmatt provides adaptive driving training. “We want people not to have to worry about if the transit system is going to show up, or whether a ride service will get them to a…

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Capricor Therapeutics, which received early funding from CureDuchenne, said the FDA has requested the full clinical study report from their latest clinical trial, the HOPE-3 trial.  Positive topline results from the HOPE-3 trial was reported in late 2025, and Capricor plans to submit the additional materials requested in February 2026.  The company expects these materials…

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The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to (Z)-endoxifen for Duchenne muscular dystrophy (DMD), providing Atossa Therapeutics with incentives to encourage further research into its DMD treatment candidate. The company said it plans to continue working closely with the FDA as it develops the once-daily oral therapy. In a company…

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I’d like to share my thoughts on what the word “strength” means. The United States has had one president who used a wheelchair: Franklin D. Roosevelt. During his time in the political spotlight, he did his best to conceal his disability. Many believe he did this to project an image of strength to the public.…

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My family and I are part of a community of boys, men, and families living with Duchenne muscular dystrophy. That community extends far beyond the U.S., where we live. Families uniquely experience Duchenne in the context of their own country, but there is a commonality in that we all want the best treatments and care.…

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