Gene therapy and other novel treatment approaches for Charcot-Marie-Tooth disease
Author:
Publisher
Elsevier BV
Subject
Genetics (clinical),Neurology (clinical),Neurology,Pediatrics, Perinatology and Child Health
Reference79 articles.
1. Ascorbic acid for Charcot-Marie-Tooth disease type 1A in children: a randomised, double blind, placebo-controlled, safety and efficacy trial;Burns;Lancet Neurol,2009
2. High dosage ascorbic acid treatment in Charcot-Marie-Tooth disease type 1A: results of a randomized, double-masked, controlled trial;Lewis;JAMA Neurol,2013
3. Ascorbic acid in Charcot-Marie-Tooth disease type 1A (CMT-TRIAAL and CMT-TRAUK): a double-blind randomised trial;Pareyson;Lancet Neurol,2011
4. Oral high dose ascorbic acid treatment for one year in young CMT1A patients: a randomised, double-blind, placebo-controlled phase II trial;Verhamme;BMC Med,2009
5. Challenges in treating charcot-marie-tooth disease and related neuropathies: current management and future perspectives;Pisciotta;Brain Sci,2021
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