Vitamin C and Charcot-Marie-Tooth 1A: Pharmacokinetic considerations

F. Visioli, M. M. Reilly, M. Rimoldi, A. Solari, D. Pareyson

Research output: Contribution to journalArticle

Abstract

Charcot-Marie-Tooth 1A disease (CMT1A) is a disease for which no drug treatments are available. In 2004, it was reported that ascorbic acid reduced the severity of neuropathy in transgenic mice overexpressing PMP22, an animal model of human CMT1A, compared with untreated mice. Based on those results, clinical trials were undertaken at different centers worldwide and four of them have been completed, but none of them resulted in significant improvements. Based on the pharmacokinetics of ascorbic acid, we propose that the randomized clinical trial carried out thus far confirmed the tight control of ascorbic acid's absorption and proved its tolerability at one and two years. The pharmacokinetic considerations discussed in this article might largely explain the disappointing results of the recent CMT1A trials.

Original languageEnglish
Pages (from-to)10-12
Number of pages3
JournalPharmaNutrition
Volume1
Issue number1
DOIs
Publication statusPublished - Jan 1 2013

Fingerprint

Charcot-Marie-Tooth Disease
Ascorbic Acid
pharmacokinetics
Tooth
teeth
Pharmacokinetics
ascorbic acid
randomized clinical trials
peripheral nervous system diseases
mice
Transgenic Mice
drug therapy
clinical trials
Randomized Controlled Trials
Animal Models
animal models
Clinical Trials
genetically modified organisms
acids
Pharmaceutical Preparations

Keywords

  • Ascorbic acid
  • Charcot-Marie-Tooth disease
  • Myelin
  • Neuromuscular disorders

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)
  • Food Science

Cite this

Vitamin C and Charcot-Marie-Tooth 1A : Pharmacokinetic considerations. / Visioli, F.; Reilly, M. M.; Rimoldi, M.; Solari, A.; Pareyson, D.

In: PharmaNutrition, Vol. 1, No. 1, 01.01.2013, p. 10-12.

Research output: Contribution to journalArticle

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