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Genetic and Rare Diseases Information Center (GARD)

Charcot-Marie-Tooth disease

Your Question

Have you heard anything regarding experimental stem cell or gene therapy for Charcot-Marie-Tooth disease? I would be interested in participating in experimental treatment. 

Our Answer

We have identified the following information that we hope you find helpful. If you still have questions, please contact us.

Are there any current research projects evaluating stem cell or gene therapy for Charcot-Marie-Tooth disease?

We were not able to find any studies that are planning to evaluate stem cell therapy or gene therapy for Charcot-Marie-Tooth disease. On our resource page for Charcot-Marie-Tooth disease, we have listed resources where you can find information on research studies and clinical trials. Check these resources often for updates.

You may also want to contact the Charcot-Marie-Tooth Association, if you have not done so already. This organization promotes research for Charcot-Marie-Tooth disease and may be aware of the latest research.

In addition, the following researcher has a study called, "Molecular Therapy and Management for Charcot-Marie-Tooth disease." You may want to contact this individual for more information on his research. The Information Center provides the names of researchers for informational purposes only and not as an endorsement of services.

James R. Lupski, M.D., Ph.D.
Department of Molecular and Human Genetics
Baylor College of Medicine
One Baylor Plaza, MS BCM225
Houston, TX, 77030, U.S.A.
Phone: 713-798-6530
Fax: 713-798-5073
Online profile: 
Last updated: 4/7/2009
Other Names for this Disease
  • CMT
  • Hereditary motor and sensory neuropathy
  • HMSN
  • Charcot Marie Tooth disease
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