A novel treatment being developed by Vanda Pharmaceuticals for a single patient with Charcot-Marie-Tooth disease type 2S (CMT2S) — an ultrarare type of this genetic disorder — has been granted rare pediatric disease designation by the U.S. Food and Drug Administration (FDA). That regulatory status aims to incentivize…
Discussion
An experimental gene therapy for Charcot-Marie-Tooth disease type 4C (CMT4C) — a rare form of the genetic disease that has muscle weakness as its hallmark symptom — was shown to improve nerve signaling, motor function, and muscle health in a mouse model. Moreover, according to the researchers, the treatment…
Becoming educated and informed about Charcot-Marie-Tooth disease 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 CMT journey.
There is no cure for CMT 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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