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Entrada Therapeutics announced it has been cleared in the U.K. to start a Phase 1/2 clinical trial of ENTR-601-44, at increasing doses, in Duchenne muscular dystrophy (DMD) patients with a mutation in the DMD gene amenable to exon 44 skipping. With this decision by U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA), which follows positive…

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Cumberland Pharmaceuticals announced positive cardiac results from Phase 2 trial in Duchenne Cumberland Pharmaceuticals released positive top-line results from its Phase 2 FIGHT DMD trial, a 12-month placebo controlled study evaluating Ifetroban in 41 individuals with DMD.  Individuals who received either low or high dose Ifetroban daily showed improvements in their heart’s left ventricular ejection…

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As an early funder of Entrada, CureDuchenne is pleased to share that Entrada received authorization in the United Kingdom (UK) to start a Phase 1/2 trial for individuals with Duchenne amenable to exon 44 skipping.  Part A, which is planned to start in Q2 2025, will be a multiple-ascending dose study in 24 individuals with…

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I read a quote the other day on Facebook: “When you are raising babies, you feel how long the days are. When raising teens, you feel with every single bone in your body how short the years are.” Days later, I still carry those words with me as a mom of seven children: Lexi, 23;…

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Upcoming Events Offer Treatment Updates and Essential Resources  to Support Families and Enhance Quality of Life  NEWPORT BEACH, Calif., January 31, 2025 – CureDuchenne, a global leader in advancing research and patient care for individuals with Duchenne and Becker muscular dystrophy, is proud to announce its 2025 schedule of events for families and caregivers. These events…

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The Muscular Dystrophy Association (MDA) has announced its 2025 Legacy Awards, and will honor two individuals who have made pioneering strides toward improving life for people affected by muscular dystrophy. The 2025 MDA Legacy Award for Achievement in Clinical Research will be given to Katherine Mathews, MD, a researcher who has studied muscular dystrophy for…

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About a month ago, I came home from playing with my church’s music group at a nursing facility. We played a doubleheader, doing a set in the memory care wing, then another in the personal care area. Because of my facioscapulohumeral muscular dystrophy (FSHD), I’ve stopped doing back-to-back performances, aside from a couple times a…

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Rediscovering my creative self has been one of the most fulfilling yet challenging parts of my journey with Duchenne muscular dystrophy (DMD). A few weeks ago, I started an exciting adventure with Wildly Creative, a 14-week arts and wellness program run by the Singapore theater company Wild Rice. Led by Edith Podesta, an Australian director…

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Two years after receiving the gene therapy Elevidys (delandistrogene moxeparvovec-rokl), motor function continues to improve in boys with Duchenne muscular dystrophy (DMD) who entered a global clinical trial able to walk. That’s according to top-line findings from part two of the Phase 3 EMBARK trial (NCT05096221), which evaluated Sarepta Therapeutics‘ approved gene therapy in more…

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Sarepta Therapeutics shared positive topline results from Part 2 of the EMBARK study, showing that treatment with the microdystrophin gene therapy, Elevidys, is associated with sustained benefits and disease stabilization in ambulatory individuals.   EMBARK is a Phase 3 study of Elevidys (in individuals aged 4-7 years) with a crossover design, in which individuals treated with…

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