Opinion statement The family of hereditary peripheral neuropathies that makes up Charcot-Marie-Tooth disease (CMT) comprises some of the most
common neuromuscular disorders. Over the past decade, understanding of the molecular basis of CMT has increased enormously.
In addition, the neurophysiologic deficits and clinical problems associated with CMT are more clearly delineated, and the
precise genetic cause of many types of CMT has now been determined. Advances in molecular biology and genetic manipulation
techniques have allowed the development of animal models of some of these CMT types, allowing more productive scientific exploration
of possible treatments. Recent treatment advances that have been effective in animal models include oral supplementation with
curcumin and vitamin C (ascorbic acid), and the use of onapristone, a progesterone antagonist. Human trials with vitamin C
are currently in progress. While ongoing molecular genetic research continues to identify more of the mutant genes and proteins
that cause the various disease subtypes, clinical research should continue to focus on developing pharmaceutical and rehabilitative
therapies to ameliorate nerve degeneration and ultimately improve function for patients with CMT. These patients optimally
should be managed in a comprehensive, multidisciplinary setting involving neurologists, physiatrists, orthopedic surgeons,
physical and occupational therapists, and orthotists. Treatment should be aimed at maximizing independence and quality of
life.
Content Type Journal ArticleDOI 10.1007/s11940-008-0011-3Authors
Gregory T. Carter, University of Washington Department of Rehabilitation Medicine Box 356490 1959 N.E. Pacific Avenue Seattle WA 98195 USAMichael D. WeissJay J. HanPhillip F. ChanceJohn D. England
Journal Current Treatment Options in NeurologyOnline ISSN 1534-3138Print ISSN 1092-8480
Journal Volume Volume 10
Journal Issue Volume 10, Number 2 / March, 2008 (Source: Current Treatment Options in Neurology)
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