I have been writing here about my family since August 2021. My husband, Jason, and I share seven children: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4. Max, Rowen, and Charlie have Duchenne muscular dystrophy (DMD). In my first column,…
Discussion
Epicrispr Biotechnologies is partnering with Forge Biologics to support the development of EPI-321, Epicrispr’s one-time epigenetic treatment candidate for facioscapulohumeral muscular dystrophy (FSHD). The treatment uses epigenetics — chemical modifications that can alter gene activity without changing a gene’s DNA sequence — to turn off the abnormally-activated…
Discussion
The death of my dear friend Timothy Chan last month still affects me deeply. A couple weeks ago, I stood at his memorial, speaking about a friend who had lived fully despite the barriers associated with a lack of awareness about Duchenne muscular dystrophy (DMD) here in Singapore.
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Regulators in the European Union have granted orphan drug designation to SGT-003, a gene therapy for Duchenne muscular dystrophy (DMD) that’s being developed by Solid Biosciences. The treatment candidate, already awarded orphan drug status in the U.S., aims to preserve muscle health in boys with DMD. This…
Discussion
My son Charlie has recently been unable to get into his power wheelchair on his own, and I know I am watching him lose the ability to ambulate, as I did with two of my other sons. Charlie, 15, like his brothers Max, 20, and Rowen, 17, lives with…
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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