シンポジウム8：チャレンジ！ 遺伝性末梢神経疾患治療
Charcot–Marie–Tooth病の治療研究の現状
2021 年 38 巻 3 号 p. 374-377
詳細
2021 年 38 巻 3 号 p. 374-377
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.
