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Becker muscular dystrophy (BMD) patients who received the experimental treatment sevasemten in clinical trials had stable motor function over several years of follow-up, new data showed. That stands in contrast to the typical progression of BMD, in which motor function steadily declines as the disease progresses. The data were presented at the Muscular Dystrophy Association…

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My facioscapulohumeral muscular dystrophy (FSHD) has progressed to the point where daily activities are increasingly challenging, and often impossible, to manage on my own. As a result, I now need more help than ever before. I know I’m doing the best I can. I exercise daily, hoping to keep myself moving for as long as…

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REGENXBIO GENE THERAPY RGX-202, an investigational gene therapy for Duchenne, continues to show a favorable safety profile with no serious adverse events, no liver injury signals, and reductions in key muscle damage biomarkers one year after treatment. Interim results from seven participants indicate improved functional performance and stable cardiac function, with strong microdystrophin expression, while…

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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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