Ascorbic acid for Charcot-Marie-Tooth disease type 1A in children: a randomised, double-blind, placebo-controlled, safety and efficacy trial.

@article{Burns2009AscorbicAF,
  title={Ascorbic acid for Charcot-Marie-Tooth disease type 1A in children: a randomised, double-blind, placebo-controlled, safety and efficacy trial.},
  author={Joshua Burns and Robert A. Ouvrier and Eppie M Yiu and Pathma D Joseph and Andrew J. Kornberg and Michael C. Fahey and Monique M. Ryan},
  journal={The Lancet. Neurology},
  year={2009},
  volume={8 6},
  pages={537-44}
}
BACKGROUND Charcot-Marie-Tooth disease type 1A (CMT1A) is the most common inherited nerve disorder. CMT1A is characterised by peripheral nerve demyelination, weakness, and impaired motor function and is caused by the duplication of PMP22, the gene that encodes peripheral myelin protein 22. High-dose ascorbic acid has been shown to have remyelinating potential and to correct the phenotype of a transgenic mouse model of CMT1A by decreasing expression of PMP22. We tested the efficacy and safety of… CONTINUE READING

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