A European Medicines Agency (EMA) committee has recommended that vamorolone be approved in the European Union to treat Duchenne muscular dystrophy (DMD) in patients ages 4 and older. The recommendation, from the EMA’s Committee for Medicinal Products for Human Use (CHMP), now will be reviewed by the European…
I always assumed my three sons with Duchenne muscular dystrophy (DMD) wouldn’t get to experience college. I knew it would depend on their health, disease progression, and openness to making it happen, as well as all the typical things like good grades and test scores. However, as my…
People with myotonic dystrophy type 1 (DM1) reported gains in measures of muscle strength after being treated with AOC 1001 in a clinical trial. That’s according to new data announced by Avidity Biosciences, the developer of AOC 1001. The findings “reinforce our belief in the potential of AOC 1001…
When I moved to Pittsburgh in January to be closer to my children and grandchildren, we were aware that we’d have to make modifications to our new home. Finding an accessible home in that city was impossible, especially during the pandemic real estate boom, when houses were selling…
The gene therapy RGX-202 at lower dose was well tolerated by three boys with Duchenne muscular dystrophy (DMD) in an early clinical trial, and biomarker data suggest the treatment is working as intended. Regenxbio, the company developing RGX-202, expects to administer a higher dose of…
My oldest son, Max, is graduating from high school this year. I know I’ve shared that repeatedly, but the long list of lasts he’s experiencing as a senior continue to inspire my writing. I have three sons with Duchenne muscular dystrophy (DMD): Max, 17, Rowen, 14, and Charlie,…
Researchers have created a three-dimensional (3D) artificial muscle model, derived from patient cells, that accurately replicates the damage observed in people with Duchenne muscular dystrophy (DMD). This new model, the scientists believe, will enable more efficient testing of treatments that could potentially reverse such muscle damage. The findings were…
Capricor Therapeutics intends to ask the U.S. Food and Drug Administration (FDA) to approve its experimental cell therapy CAP-1002 for treating Duchenne muscular dystrophy (DMD) in 2025. The company recently completed a meeting with the agency, which confirmed that data from the ongoing Phase 3 clinical…
I was mad at my husband, Jason, for spending the night in the recliner. He’d been there because he’d been snoring loudly enough at night that I hadn’t been sleeping well, and he wanted to ensure I got a good night’s sleep. But I was in no mood to recognize…
A Phase 2 clinical trial testing the investigational oral medication EDG-5506 in people with Becker muscular dystrophy (BMD) is expanding, with plans to enroll 120 additional adults with the genetic disorder. According to EDG-5506’s developer Edgewise Therapeutics, enrollment in the expanded study is ongoing. The company is hosting…
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