The experimental gene therapy Engensis (VM202) was found to be safe and showed early signs of benefit for people with Charcot-Marie-Tooth disease type 1A (CMT1A), according to results from a small Phase 1/2a clinical trial. Treatment reduced disease severity, mainly by easing sensory neuropathy, or nerve damage affecting sensation, and lessened…
Discussion
Second in a series. Read part one. Last year, the world lost a powerful voice in the disability community with the death of Alice Wong. Born with a type of spinal muscular atrophy (SMA), her life was not given much attention by mainstream media, and her ideas…
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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