March 2026

REGENXBIO has announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202, an investigational gene therapy being developed for individuals with Duchenne muscular dystrophy. RGX-202 is designed to deliver microdystrophin via AAV8 through a one-time IV infusion. In the interim functional results from seven participants treated at the pivotal dose level…

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Solid Biosciences Inc. has shared updated positive interim data from 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 AAV capsid, which is designed to increase skeletal and cardiac muscle targeting while detargeting the liver. According…

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ITF shared a community letter and presented new data, including long-term safety observations based on data from the company’s ongoing open-label extension study in patients with Duchenne muscular dystrophy treated with givinostat. In addition, analyses from the givinostat clinical development program include findings on reduction of decline in muscle contractile area and potential delay in median age at loss of…

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SGT-003, an investigational microdystrophin gene therapy for Duchenne, continues to demonstrate an encouraging safety profile and has been generally well tolerated in 40 participants treated to date. Interim data show robust microdystrophin expression, restoration of key components of the dystrophin-associated protein complex (DAPC), improvements in biomarkers of muscle integrity, and stabilization or improvement in cardiac…

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Living with a rare disease is incredibly complex, with countless layers to navigate. Managing daily life amid so many unknowns — and trying to find a regimen that truly works — can feel overwhelming. From the routine of medications and other relentless therapies to countless medical frustrations, we’re forced to become experts in our own…

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Capricor Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has lifted the July 2025 Complete Response Letter and resumed review of the company’s Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for the treatment of Duchenne cardiomyopathy. The FDA has assigned a new Prescription Drug User Fee Act (PDUFA) target…

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At this year’s Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, researchers and clinicians are talking about the central role of patients, families, and caregivers in driving change for people living with neuromuscular diseases. Muscular Dystrophy News Today sat down with John F. Crowley, president and CEO of the Biotechnology Innovation Organization (BIO) and this…

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As an early funder of Capricor Therapeutics, CureDuchenne is pleased to share that the FDA has set the PDUFA target action date for August 22, 2026 as the deadline to review approval of deramiocel for Duchenne muscular dystrophy.  Deramiocel is a cell therapy that exerts immunomodulatory and anti-fibrotic actions on cardiac and skeletal muscle.   Read…

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Edgewise Therapeutics, a CureDuchenne funded company, today announced positive long-term Sevasemten data that demonstrated sustained functional stabilization in Becker Muscular Dystrophy (BMD) patients through 3.5 years of treatment. This result is in stark contrast to the functional decline expected from BMD natural history data. Today’s result is particularly important for BMD patients, an underserved population,…

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Edgewise Therapeutics, Inc. has shared long-term data from its MESA open-label extension study of sevasemten in Becker muscular dystrophy. Sevasemten is an orally administered small molecule inhibitor designed to protect muscle against contraction-induced damage in muscular dystrophies. MESA is an open-label extension evaluating sevasemten long-term safety, tolerability, and efficacy in adults and adolescents with Becker…

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