High-dosage ascorbic acid treatment in Charcot-Marie-Tooth disease type 1A: results of a randomized, double-masked, controlled trial
RA Lewis, MP McDermott, DN Herrmann… - JAMA …, 2013 - jamanetwork.com
Importance No current medications improve neuropathy in subjects with Charcot-Marie-
Tooth disease type 1A (CMT1A). Ascorbic acid (AA) treatment improved the neuropathy of a
transgenic mouse model of CMT1A and is a potential therapy. A lower dosage (1.5 g/d) did
not cause improvement in humans. It is unknown whether a higher dosage would prove
more effective. Objective To determine whether 4-g/d AA improves the neuropathy of
subjects with CMT1A. Design A futility design to determine whether AA was unable to …
Tooth disease type 1A (CMT1A). Ascorbic acid (AA) treatment improved the neuropathy of a
transgenic mouse model of CMT1A and is a potential therapy. A lower dosage (1.5 g/d) did
not cause improvement in humans. It is unknown whether a higher dosage would prove
more effective. Objective To determine whether 4-g/d AA improves the neuropathy of
subjects with CMT1A. Design A futility design to determine whether AA was unable to …