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Charcot-Marie-Tooth Disease
Charcot-Marie-Tooth (CMT) disease is a group of inherited, genetic disorders that affect the nerves. This type of nerve problem is called hereditary neuropathy. That means it is passed from parents to children.
CMT disease affects both the sensory and motor nerves that connect the brain to the rest of the body. These nerves are called peripheral nerves. Peripheral nerves include:
- Motor nerves: help you move muscles
- Sensory nerves: help you feel sensations such as hot, cold, and vibrations
CMT disease is most often caused by inherited gene changes (called gene mutations). There are different gene changes linked to CMT. Gene changes can include:
- Damage to the fibers within nerves, called the axon
- Damage to the protective coating around the nerves, called the myelin sheath
Both kinds of gene changes cause the nerves to be damaged. This damage affects the way information travels through the nerve cells.
To learn more about CMT and current treatments, visit organizations dedicated to CMT and rare diseases.
Charcot-Marie-Tooth disease care at St. Jude
St. Jude Children’s Research Hospital is working to deepen the understanding of rare, catastrophic genetic neurological disorders in children. Our aim is to transform the treatment landscape for these disorders. The Center for Experimental Neurotherapeutics (CENT) was established at St. Jude as part of the Pediatric Transitional Neuroscience Initiative (PTNI) to focus on these neurological disorders.
Select children with CMT who are accepted at St. Jude take part in clinical research or clinical trials. The aim of these studies is to help scientists learn more about the disorders and to develop potential treatments.
Charcot-Marie-Tooth disease clinical trials
St. Jude offers clinical trials and cancer research studies for children, teens, and young adults for Charcot-Marie-Tooth disease.
Recruiting
Study goal:
This long-term study aims to better understand the genetic causes of Charcot-Marie-Tooth disease (CMT) and to learn how different forms of the rare hereditary disease affect people over time.
Age:
Birth to 22 years
Seeking treatment at St. Jude
Patients accepted to St. Jude must have a disease we treat and must be referred by a physician or other qualified medical professional. We accept most patients based on their ability to enroll in an open clinical trial.
Contact the Physician / Patient Referral Office
Call: 1-888-226-4343 (toll-free) or 901-595-4055 (local) | Fax: 901-595-4011 | Email: referralinfo@stjude.org | 24-hour pager: 1-800-349-4334