Dosing has begun in a Phase 2 clinical trial testing the oral therapy SAT-3247 in boys with Duchenne muscular dystrophy (DMD). The study, dubbed BASECAMP (NCT07287189), aims to enroll 51 boys with DMD aged 7-9 who can walk. Patients who have received treatments such as exon skippers,…
Discussion
I was born in March 1988 with the umbilical cord wrapped around my ankles three times. My heart rate dropped to zero, and the doctors had to do an emergency cesarean section to save me. I still reached all my milestones like a normal baby. When I was 6 years…
Discussion
It’s been a busy couple of weeks. There have been lots of appointments and activities, and I’ve been busy running errands around town. I live in a rural community in south central Nebraska. It’s small enough that, no matter where I go, I run into someone I know. I also…
The U.S. Food and Drug Administration (FDA) has cleared Precision Biosciences to launch a clinical trial testing its gene-editing therapy candidate PBGENE-DMD in people with Duchenne muscular dystrophy (DMD) caused by certain mutations. Precision announced it had received a “Study May Proceed notification” from the U.S. regulatory agency…
Discussion
Pittsburgh, where I now live, receives far less annual snowfall than Shanksville, Pennsylvania, where my wife, Wendy, and I lived for 41 years. Shanksville, in Somerset County, sits squarely in the state’s snow belt. However, every so often, Pittsburgh experiences true winter weather, with bitter cold and significant snowfall. Just…
Becoming educated and informed about muscular dystrophy is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnists’ words of wisdom to help you along on your muscular dystrophy journey.
There is no cure for muscular dystrophy yet, but here you can find more information about current therapeutic approaches, as well as experimental treatments that are in the pipeline.
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