Rett Syndrome

A 12-month, double-blind, placebo-controlled folate–betaine trial studied 68 females with Rett syndrome. Participants were randomized as young (< age 5 years) or old ( age 5 years). Structured clinical assessments occurred at baseline, 3, 6, and 12 months. Primary outcome measures included quantitative evaluation of breathing and hand movements during wakefulness, growth, anthropometry, motor/behavioral function, and qualitative evaluations from electroencephalograms and parent questionnaires.

Objective evidence of improvement was not found, but subjective improvement from parent questionnaires was noted for the <5 years group.

Glaze et al (2009). "A Study of the Treatment of Rett Syndrome With Folate and Betaine." J Child Neurol. Feb 18 epub.