Charcot-Marie-Tooth-Disease (cont.)

What research is being done?

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.

Where can I get more information?

For more information on neurological disorders or research programs funded by the National Institute of Neurological Disorders and Stroke, contact the Institute's Brain Resources and Information Network (BRAIN) at:

BRAIN P.O. Box 5801
Bethesda, MD 20824
(800) 352-9424

Information also is available from the following organizations:

Charcot-Marie-Tooth Association (CMTA)
2700 Chestnut Parkway Chester, PA 19013-4867
Tel: 610-499-9264 800-606-CMTA (2682) Fax: 610-499-9267

Muscular Dystrophy Association
National Office - 222 S. Riverside Plaza
Suite 1500 Chicago, IL 60606
Tel: 800-572-1717 Fax: 520-529-5300

Neuropathy Association
110 West 40th Street Suite 1804
New York, NY 10018
Tel: 888-PN-FACTS (888-763-2287) Fax: 212-692-0668

Hereditary Neuropathy Foundation, Inc
432 Park Avenue South 4th Floor
New York, NY 10128
Tel: 855-HELPCMT (435-7268) 212-722-8396


US National Institute of Neurological Disorders and Stroke. "Charcot-Marie-Tooth Disease Fact Sheet." Last updated: 2/23/2015

Medically Reviewed by a Doctor on 2/23/2015

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