What is the role of medications in the treatment of Charcot-Marie-Tooth disease (CMT)?

Updated: Jun 23, 2021
  • Author: Divakara Kedlaya, MBBS; Chief Editor: Elizabeth A Moberg-Wolff, MD  more...
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Avoid drugs and medications known to cause nerve damage (eg, vincristine, [82, 83] isoniazid, nitrofurantoin). Identify the cause of any pain as accurately as possible. Musculoskeletal pain may respond to acetaminophen or nonsteroidal anti-inflammatory drugs (NSAIDs). Neuropathic pain may respond to tricyclic antidepressants or antiepileptic drugs, such as carbamazepine or gabapentin.

Dyck and colleagues [84] and Ginsberg and coauthors [85] described a few individuals with Charcot-Marie-Tooth disease type 1 (CMT-1) and sudden deterioration in whom treatment with steroids (prednisone) or intravenous immunoglobulin produced variable levels of improvement.

Sahenk and colleagues had studied the effects of neurotrophin-3 (NT3), a neurotrophic factor, on individuals with CMT-1A. It was known to promote axonal growth and was tested with favorable results in 2 animal models and in a pilot study involving 8 CMT-1A patients. [86, 87]

Passage and coauthors [88] reported therapeutic benefits from the administration of ascorbic acid (vitamin C) in a mouse model of CMT-1. Based on these results, clinical trials were undertaken at different centers worldwide, and 4 of them have been completed; unfortunately, however, none of them resulted in significant clinical improvements. [89, 90, 91, 92]

The progesterone antagonist onapristone proved to be effective in a rat model of CMT-1A; unfortunately, currently available progesterone antagonists are too toxic to be safely used in humans. [93, 94, 95]

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