Journal article
Current therapy for Charcot-Marie-Tooth disease
Current treatment options in neurology, Vol.7(1), pp.23-31
01/2005
DOI: 10.1007/s11940-005-0003-5
PMID: 15610704
Abstract
Charcot-Marie-Tooth (CMT), or heritable peripheral neuropathies, is among the most frequent genetic neuromuscular disorders, with a prevalence of approximately 1:2500. Since 1991, remarkable advances have occurred in determining the precise genetic cause of many forms of CMT and in generating animal models of many of these disorders. However, these advances have not yet resulted in cures for CMT. Recently, potential treatments for the most common form of CMT, CMT-1A, have been shown in rodent models of the disorder. Treatment with onapristone, a progesterone antagonist, has improved the neuropathy of the CMT-1A rat. Treatment with large doses of ascorbic acid (vitamin C) has improved the neuropathy of the CMT-1A mouse. Multicentric trials with ascorbic acid are likely to start in the near future to assess if vitamin C supplementation is effective and what is the dosage required in humans to improve neurologic disability. Because of potential side effects with antiprogesterone therapy, particularly in women of childbearing age, research is actively proceeding with progesterone antagonists to develop safe medications that also can be used in clinical trials of CMT-1A. Although no cures are available for CMT, there are many important treatments available for patients with CMT that can improve their quality of life and help them maintain their independence. Some of these therapies involve physiatry and orthopedic surgery. Others involve pain management. Lastly, there are potential concerns about medications or lifestyle issues that may exacerbate CMT. All of these issues will be discussed.
Details
- Title: Subtitle
- Current therapy for Charcot-Marie-Tooth disease
- Creators
- Marina GrandisMichael Shy - Department of Neurology Wayne State University 4201 Saint Antoine Street 48201 Detroit MI USA
- Resource Type
- Journal article
- Publication Details
- Current treatment options in neurology, Vol.7(1), pp.23-31
- Publisher
- Current Medicine Group; Philadelphia
- DOI
- 10.1007/s11940-005-0003-5
- PMID
- 15610704
- ISSN
- 1092-8480
- eISSN
- 1534-3138
- Language
- English
- Date published
- 01/2005
- Academic Unit
- Neurology; Molecular Physiology and Biophysics; Stead Family Department of Pediatrics; Iowa Neuroscience Institute
- Record Identifier
- 9984020896302771
Metrics
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