Joëlle Micallef, Shahram Attarian, Odile Dubourg, Pierre-Marie Gonnaud, Jean-Yves Hogrel, Tanya Stojkovic, Rafaelle Bernard, Elisabeth Jouve, Severine Pitel, Francois Vacherot, Jean-Francois Remec, Laurent Jomir, Eric Azabou, Mahmoud Al-Moussawi, Marie-Noelle Lefebvre, Laurence Attolini, Sadek Yaici, Daniel Tanesse, Michel Fontes, Jean Pouget, Olivier Blin
BACKGROUND: Charcot-Marie-Tooth disease type 1A (CMT1A) is a hereditary peripheral neuropathy that affects roughly one in 5000 births. No specific therapy currently exists for this degenerative disorder, which is characterised by distal progressive muscle atrophy and sensory loss, although ascorbic acid has been shown to reduce demyelination and improve muscle function in a transgenic mouse model of CMT1A. We tested the safety and efficacy of ascorbic acid in adults with CMT1A. METHODS: This 12-month, randomised, double-blind, placebo-controlled study was undertaken between September, 2005, and October, 2008...
December 2009: Lancet Neurology