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Charcot-Marie-Tooth-Disease (cont.)

What research is being done on Charcot-Marie-Tooth disease?

The NINDS supports research on CMT and other peripheral neuropathies in an effort to learn how to better treat, prevent, and even cure these disorders. Ongoing research includes efforts to identify more of the mutant genes and proteins that cause the various disease subtypes, efforts to discover the mechanisms of nerve degeneration and muscle atrophy with the hope of developing interventions to stop or slow down these debilitating processes, and efforts to find therapies to reverse nerve degeneration and muscle atrophy.

One promising area of research involves gene therapy experiments. Research with cell cultures and animal models has shown that it is possible to deliver genes to Schwann cells and muscle. Another area of research involves the use of trophic factors or nerve growth factors, such as the hormone androgen, to prevent nerve degeneration. Vitamin C has been studied in CMT1A and the results of a multicentric trial are due soon. Curcumin, a component of curry, is currently being studied as a treatment strategy in an animal model of CMT1B.

Medically reviewed by Edward Spence, MD; American Board of Pediatrics and American Board of Medical Genetics with subspecialties in Clinical Genetics, Clinical Biochemical Genetics, Clinical Molecular Genetics

Last updated: 5/12/2010 SOURCE:

"Charcot-Marie-Tooth Disease Fact Sheet." National Institute of Neurological Disorders and Stroke. 8 July 2013.

Medically Reviewed by a Doctor on 4/2/2014


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