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Each year, the Duchenne and Becker muscular dystrophy community comes together in Washington, DC with a shared mission: to ensure that the voices of individuals living with Duchenne and Becker and their families are heard where policy decisions are made. This year, PPMD’s Advocacy Conference once again demonstrated the extraordinary power of community advocacy. Families,…

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PGN-EDODM1, Pepgen’s experimental therapy for myotonic dystrophy type 1 (DM1), was generally well tolerated in an early clinical trial, with biomarker data suggesting that the treatment is affecting its intended molecular target.  Based on these early findings, Pepgen is now sponsoring a Phase 2 trial, FREEDOM2-DM1 (NCT06667453), to evaluate the safety of repeated doses of…

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Capricor Therapeutics has announced additional analyses and new functional outcomes data from the Phase 3 HOPE-3 clinical trial of Deramiocel in Duchenne. Deramiocel is the company’s investigational cell therapy for the treatment of Duchenne cardiomyopathy. Cardiac MRI analyses demonstrated Deramiocel’s impact on the heart, with data showing reduction in fibrotic segments in patients treated with…

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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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