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- W2060175036 abstract "In this issue of The Lancet Neurology, Burns and colleagues 1 Burns J Ouvrier RA Yiu EM et al. Ascorbic acid for Charcot–Marie–Tooth disease type 1A in children: a randomised, double-blind, placebo-controlled safety and efficiency trial. Lancet Neurol. 2009; (published online May 7.)https://doi.org/10.1016/S1474-4422(09)70108-5 Summary Full Text Full Text PDF Scopus (114) Google Scholar report the results of their randomised controlled trial of children with Charcot–Marie–Tooth disease type 1A (CMT1A) who were given ascorbic acid over 12 months. These results, and those of similar trials in adults, have been eagerly awaited for at least two reasons. First, there is no effective medication to cure or decrease the progression of any form of CMT disease. Second, and perhaps as important, these trials mark the beginning of a new era for inherited neuropathies, in which the development of rational treatments is based on knowledge of the genetics and biology of the disease. Ascorbic acid for Charcot–Marie–Tooth disease type 1A in children: a randomised, double-blind, placebo-controlled, safety and efficacy trial12 months of treatment with high-dose ascorbic acid was safe and well tolerated but none of the expected efficacy endpoints were reached. Full-Text PDF" @default.
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- W2060175036 date "2009-06-01" @default.
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- W2060175036 title "Ascorbic acid for treatment of CMT1A: the jury is still out" @default.
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- W2060175036 doi "https://doi.org/10.1016/s1474-4422(09)70112-7" @default.
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