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Cumberland Pharmaceuticals Announces Positive Results from Phase 2 FIGHT DMD Trial of Ifetroban for Treatment of Duchenne Cardiomyopathy
Cumberland Pharmaceuticals Inc. has announced positive top-line results from its Phase 2 FIGHT DMD trial evaluating ifetroban for the treatment of Duchenne cardiomyopathy. Ifetroban is an oral thromboxane receptor antagonist aimed at reducing inflammation and fibrosis to protect cardiac function, and has received both Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA.…
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Exploring new possibilities in the realm of art and disability
The Lunar New Year has always been a time of reflection and renewal for me. This year, though, it was bittersweet because my longtime caregiver plans to return to her home country in a few weeks. With her departure on the horizon, I scaled back visits to extended family during this festive period. My caregiver’s…
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Phase 1/2 trial of exon 44 skipping therapy for DMD cleared in UK
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 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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Entrada Therapeutics gets ok to start Exon 44 skipping trial in UK
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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To a parent, the years always seem too short
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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CureDuchenne Announces Educational Events for Families and Caregivers of Individuals with Duchenne or Becker Muscular Dystrophy
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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MDA honors researcher, patient advocate with 2025 Legacy Awards
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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Another fall left me feeling like an upside-down turtle
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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How a Singapore ‘Artist’s Way’ program is rekindling my creativity
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…
