Noto Yu-ichi
Department of Neurology, Graduate School of Medical Science, Kyoto Prefectural University of Medicine.
Brain Nerve. 2015 Oct;67(10):1241-6. doi: 10.11477/mf.1416200289.
Charcot-Marie-Tooth disease type 1A (CMT1A) is a disease for which no drug treatments are available. Passage et al. reported that ascorbic acid reduced the mRNA level of PMP22, improved motor function and increased the numbers of myelinated peripheral nerve axons in a mouse model of CMT1A. Based on these results, five clinical trials were undertaken at different centers worldwide. However, none of them demonstrated significant effectiveness. Although these outcomes were disappointing, these studies have provided many useful insights for conducting the next randomised controlled trial for CMT1A.
1型遗传性运动感觉神经病（CMT1A）是一种尚无药物治疗的疾病。帕西等人报告称，在CMT1A小鼠模型中，抗坏血酸降低了PMP22的mRNA水平，改善了运动功能，并增加了有髓外周神经轴突的数量。基于这些结果，全球不同中心开展了五项临床试验。然而，这些试验均未显示出显著疗效。尽管这些结果令人失望，但这些研究为开展下一项CMT1A随机对照试验提供了许多有用的见解。
