On the cover: TyKiah Wright of Columbus, Ohio, has been affected by CMT since childhood. Now 31, she earned bachelor’s and MBA degrees from Wright State University in Dayton. In 2001, she established the WrightChoice Intern Program, which helps minority students and students with disabilities find internships and jobs.

Dear Friends What is Charcot-Marie-Tooth Disease? What causes CMT? What happens to someone with CMT, and how is it treated? What are the different types of CMT? How is CMT diagnosed? Does it Run in the Family? MDA’s Search for Treatments & Cures MDA is Here to Help You

MDA’s Search for Treatments & Cures

In 1991, the genetic causes of CMT were completely unknown. But just 10 years later, MDA-funded scientists had helped to identify 10 CMT-linked genes and found evidence for several others. (There are now thought to be some 30 genes in which flaws can cause CMT.) This accomplishment has led to genetic testing for many types of CMT, which has greatly improved diagnosis.

Of equal importance, the ongoing hunt for CMT genes has given insights into treatments that might be used to stop or reverse the disorder. As the CMT gene hunt continues, MDA-funded scientists are investigating how and why specific genetic mutations lead to different types of CMT. In the future, this knowledge could enable physicians to more accurately predict the course of CMT in individual patients.

In addition to genetic advances, MDAfunded scientists have made significant progress in understanding the biology of axons and Schwann cells — the cells that make myelin in the peripheral nerves.

Some MDA-supported groups are studying myelin formation, with the aim of developing remyelination therapies for CMT.

Others are concentrating on understanding how various CMT-causing genetic mutations affect the way molecules are transported in CMT-affected and unaffected nerve cells and between nerve cells and the spaces around them.

One scientist is conducting laboratory experiments to see whether a compound called heat shock protein 90 might be therapeutic in the type 1A form of CMT, while others are conducting an MDAfunded clinical trial to test the effects of high-dose ascorbic acid on CMT1A.

In addition to these specific projects, MDA is supporting the CMT North American Database, a secure repository of information about CMT provided by patients and families. (See http://neurology.med.wayne.edu/neurogenetics/na_database.php.) MDA also is supporting the North American CMT Network, an extension to the Database designed to provide an infrastructure for CMT research.

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