A Phase 1/2 clinical trial testing SAR446268, an experimental gene therapy for myotonic dystrophy type 1 (DM1), is enrolling patients. The trial, dubbed BrAAVe (NCT06844214), is expected to enroll approximately 32 DM1 patients aged 10-50. The study is actively recruiting participants at sites in Florida, New York,…
Living with Duchenne muscular dystrophy means every trip to the hospital is key to my survival. From heart scans to routine medical appointments, leaving home is never simple. I rely on my mom to ferry me to every checkup in our family’s wheelchair-accessible vehicle. Earlier this year, when…
In recognition of Duchenne Muscular Dystrophy Awareness Month in September, the Duchenne Muscular Dystrophy Community Spotlight campaign features a series of stories highlighting the real-life experiences of people affected by Duchenne muscular dystrophy, written in their own words. Follow us on Facebook, Instagram, X, and Pinterest…
In recognition of Duchenne Muscular Dystrophy Awareness Month in September, the Duchenne Muscular Dystrophy Community Spotlight campaign features a series of stories highlighting the real-life experiences of people affected by Duchenne muscular dystrophy, written in their own words. Follow us on Facebook,…
A combined regimen of testosterone and growth hormone led to significant improvements in muscle mass, strength, and mobility in men with facioscapulohumeral muscular dystrophy (FSHD) in a clinical trial. The Phase 1/2 STARFISH trial (NCT03123913) evaluated the combination therapy’s safety, tolerability, and potential efficacy in men with FSHD.
In recognition of Duchenne Muscular Dystrophy Awareness Month in September, the Duchenne Muscular Dystrophy Community Spotlight campaign features a series of stories highlighting the real-life experiences of people affected by Duchenne muscular dystrophy, written in their own words. Follow us on Facebook, Instagram, X, and Pinterest…
I expected today, as this column is published, to be long and exhausting. I’d been scheduled to drive to my central Nebraska home from Denver, then help the marching band perform at halftime of the high school football game. I’m a mom to seven children: Lexi, 24; Max, 19;…
In recognition of Duchenne Muscular Dystrophy Awareness Month in September, the Duchenne Muscular Dystrophy Community Spotlight campaign features a series of stories highlighting the real-life experiences of people affected by Duchenne muscular dystrophy, written in their own words. Follow us on Facebook, Instagram, X, and Pinterest…
Long-term treatment with Duvyzat (givinostat) delays the loss of key motor skills in people with Duchenne muscular dystrophy (DMD), published results from an open-label extension study show. “The sustained benefit observed across functional outcomes reinforces the potential of Duvyzat to meaningfully alter the course of the disease,” Scott…
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