Ascorbic Acid in Charcot-Marie-Tooth Disease

J Millichap

doi:10.15844/pedneurbriefs-23-12-11

Author:

J Gordon Millichap

Northwestern University Feinberg School of Medicine, US

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Abstract

Ascorbic acid has been shown to reduce demyelination and improve muscle function in a transgenic mouse model of Charcot-Marie-Tooth disease (CMT1A).

How to Cite: Millichap, J.G., 2009. Ascorbic Acid in Charcot-Marie-Tooth Disease. Pediatric Neurology Briefs, 23(12), pp.96–96. DOI: http://doi.org/10.15844/pedneurbriefs-23-12-11

Ascorbic acid has been shown to reduce demyelination and improve muscle function in a transgenic mouse model of Charcot-Marie-Tooth disease (CMT1A). Aberrant expression of the myelin protein 22 gene, PMP22 is the cause of CMT1A, and large doses of ascorbic acid are shown to inhibit cAMP-mediated stimulation of human PMP22 expression. A 12-month, randomized, double-blind, placebo-controlled study of ascorbic acid in 117 adult patients compared to 62 receiving placebo found no significant difference between groups in neuropathy scores. Doses of ascorbic acid were 1 g and 3 g daily. The occurrence of adverse events did not differ between groups. [1]

COMMENT. Similar negative results were obtained in a placebo-controlled trial of ascorbic acid (30 mg/kg/day) in 81 children with CMT1A (2-16 years of age). [2]

References

Micallef, J Attarian, S Dubourg, O Gonnaud, PM Hogrel, JY Stojkovic, T et al. (2009). Effect of ascorbic acid in patients with Charcot-Marie-Tooth disease type 1A: a multicentre, randomized, double-blind, placebo-controlled trial. Lancet Neurol Dec 20098(12): 1103–1110, DOI: https://doi.org/10.1016/S1474-4422(09)70260-1 [PubMed]

Burns, J Ouvrier, RA Yiu, EM Joseph, PD Kornberg, AJ Fahey, MC et al. (2009). Ascorbic acid for Charcot-Marie-Tooth disease type 1A in children: a randomised, double-blind, placebo-controlled, safety and efficacy trial. Lancet Neurol Jun 20098(6): 537–44, DOI: https://doi.org/10.1016/S1474-4422(09)70108-5 [PubMed]

[CIT0001] Micallef, J Attarian, S Dubourg, O Gonnaud, PM Hogrel, JY Stojkovic, T et al. (2009). Effect of ascorbic acid in patients with Charcot-Marie-Tooth disease type 1A: a multicentre, randomized, double-blind, placebo-controlled trial. Lancet Neurol Dec 20098(12): 1103–1110, DOI: https://doi.org/10.1016/S1474-4422(09)70260-1 [PubMed]

[CIT0002] Burns, J Ouvrier, RA Yiu, EM Joseph, PD Kornberg, AJ Fahey, MC et al. (2009). Ascorbic acid for Charcot-Marie-Tooth disease type 1A in children: a randomised, double-blind, placebo-controlled, safety and efficacy trial. Lancet Neurol Jun 20098(6): 537–44, DOI: https://doi.org/10.1016/S1474-4422(09)70108-5 [PubMed]

Reading: Ascorbic Acid in Charcot-Marie-Tooth Disease

Neuromuscular Disorders

Ascorbic Acid in Charcot-Marie-Tooth Disease

Author:

J Gordon Millichap

Northwestern University Feinberg School of Medicine, US

X close

Abstract

References