Current research on the treatment of Charcot–Marie–Tooth disease

Abstract

Ascorbic acid, curcumin, neurotrophin 3, and neuregulin 1 have been investigated for the treatment of Charcot–Marie–Tooth disease (CMT), but none have shown efficacy. Recently, PXT3003, a fixed–dose combination of baclofen, naltrexone, and D–sorbitol, has been attracting attention. The drug is believed to reduce the expression of overexpressed PMP22 mRNA. Phase III studies have been conducted mainly in Europe, and the Overall Neuropathy Limitations Scale (ONLS) evaluation has shown efficacy as well as safety, and future reports are expected. Finally, we will discuss HDAC6 inhibitors as potential therapeutic agents for axonal CMT.