title: [Ascorbic Acid and Charcot-Marie-Tooth Disease].
abstract: 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.
authors.affiliation: Department of Neurology, Graduate School of Medical Science, Kyoto Prefectural University of Medicine.
authors.forename: Yu-ichi
authors.initials: Y
authors.lastname: Noto
mesh_terms: D000818:Animals; D001205:Ascorbic Acid / Q000627:therapeutic use*; D002607:Charcot-Marie-Tooth Disease / Q000188:drug therapy* / Q000235:genetics; D002986:Clinical Trials as Topic; D006801:Humans; D011506:Proteins / Q000235:genetics
chemical_list: C109561:CDRT1 protein, human; D011506:Proteins; D001205:Ascorbic Acid