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Solid Biosciences Shares Initial Data from INSPIRE DUCHENNE Study of Gene Therapy Candidate SGT-003
Solid Biosciences Inc. has announced positive initial data from the company’s Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003. SGT-003 is a gene therapy candidate for the treatment of Duchenne that delivers a microdystrophin via their novel AAV capsid designed to increase skeletal and cardiac muscle targeting while detargeting the liver. Solid shared interim 90-day biopsy…
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MD trial update: 5 OPMD patients safely treated with gene therapy
Five people with oculopharyngeal muscular dystrophy (OPMD) — a type of muscular dystrophy marked by muscle weakness affecting the face and throat — have thus far been treated with the gene therapy candidate BB-301 in an ongoing clinical trial, treatment developer Benitec Biopharma said in a company update. According to Benitec, the fourth and fifth…
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A week in the life of an overwhelmed caregiver
I’ll be straightforward about caregiving: It’s exhausting. I’m the mother of seven children: Lexi, 23, Max, 19, Chance, 17, Rowen, 16, Charlie, 14, Mary, 10, and Callie, 3. I’m also a caregiver to Max, Rowen, and Charlie, who have Duchenne muscular dystrophy (DMD). You are catching me during a challenging two-week period. My husband, Jason,…
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Phase 1 trial of SAT-3247 finishes enrolling healthy volunteers
A Phase 1 clinical trial testing SAT-3247, an oral treatment candidate for Duchenne muscular dystrophy (DMD), is now done enrolling healthy volunteers, its developer Satellos Bioscience announced. “The completion of healthy volunteer enrollment in our Phase 1 trial marks a significant milestone in our mission to develop transformative therapies for patients with degenerative diseases,” Frank…
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It started with shoes, but now everything is too heavy
One thing I was born with, aside from my facioscapulohumeral muscular dystrophy (FSHD), was narrow feet. As part of a working-class family, where money always seemed to be in short supply, my feet became an added expense that my mom combated by being a thrifty shopper. If she saw shoes on sale that I’d eventually…
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World of Warcraft event raises $2M to support DMD research, care
A fundraiser through the online role-playing game World of Warcraft has raised more than $2 million to support CureDuchenne, an organization dedicated to combating Duchenne muscular dystrophy (DMD). The fundraiser, which launched last year in partnership with Warcraft’s maker Blizzard Entertainment, is the largest contribution from a corporation given to CureDuchenne, according to a press…
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Growing into limb-girdle muscular dystrophy — and, better, marriage
I’ve been married for 11 years. Before then, as I grew up with what I eventually discovered was limb-girdle muscular dystrophy, I learned a lot about life, perseverance, and relationships. During my 20s, I had long relationships with a couple of women with whom I thought I’d possibly spend my life. Also throughout that decade, I…
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Blizzard Entertainment® Raises Over $2 Million for CureDuchenne Through World of Warcraft ® Charity Pet Program, Funding Lifesaving Duchenne Muscular Dystrophy Research
Inspired by “The Remarkable Life of Ibelin” documentary, Players Around the World Uniteto Honor Mats Steen and Drive Groundbreaking Research and Vital Care NEWPORT BEACH, California (February 5, 2025) – CureDuchenne, the leading nonprofit dedicated to finding a cure for Duchenne muscular dystrophy, proudly announces the overwhelming success of a recent charity partnership with Blizzard…
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Napa in Newport Returns for Its 11th Annual Celebration of Wine, Culinary Excellence, and Philanthropy
Southern California’s Premier Wine Gala Set for March 1, 2025, at Pendry Newport Beach NEWPORT BEACH Calif., January 30, 2025– Esteemed for its unparalleled showcase of Napa Valley’s finest wines, Napa in Newport returns for its 11th annual gala on March 1, 2025, at the luxurious Pendry Newport Beach. This year’s event promises an elevated…
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Phase 2 trial shows DMD treatment ifetroban boosts heart function
Treatment with oral therapy ifetroban led to improvements in measures of heart function in people with Duchenne muscular dystrophy (DMD), according to top-line results from a clinical trial. The “impressive results” are a “pivotal moment” for developer Cumberland Pharmaceuticals, “and, more importantly, for the DMD community,” A.J. Kazimi, CEO of Cumberland, said in a company…
