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…
A Phase 3 trial testing SGT-003, a gene therapy candidate for Duchenne muscular dystrophy (DMD), will start dosing before the end of March, according to Solid Biosciences. The trial follows a successful meeting with the U.S. Food and Drug Administration (FDA), during which the company and the agency aligned…
Discussion
We all have bad days, but hopefully they are few and far between. Bad days can overwhelm, dampen your spirit, and leave you feeling angry, sad, or depressed. You may need to take a break from whatever has caused them. First, let me clarify that I am talking about bumps…
Discussion
When the germs of cold and flu season find their way into the Vertin household, they crash our party of nine, and no one is spared. A cold or flu can be especially dangerous for my three sons with Duchenne muscular dystrophy (DMD) — Max, Rowen, and Charlie…
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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