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A 12-month, double-blind, placebo-controlled folate\u2014betaine trial enrolled 73 methylCpG-binding protein 2 mutation positive female participants meeting consensus criteria for Rett syndrome. Participants were randomized as young (&lt; 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. In all, 68 participants completed the study. Objective evidence of improvement was not found. Subjective improvement from parent questionnaires was noted for the &lt;5 years group. This study should inform future treatment trials regarding balancing participants with specific mutations and comparable severity to minimize selection bias.<\/jats:p>","DOI":"10.1177\/0883073808327827","type":"journal-article","created":{"date-parts":[[2009,2,18]],"date-time":"2009-02-18T21:53:17Z","timestamp":1234993997000},"page":"551-556","source":"Crossref","is-referenced-by-count":34,"title":["A Study of the Treatment of Rett Syndrome With Folate and Betaine"],"prefix":"10.1177","volume":"24","author":[{"given":"Daniel G.","family":"Glaze","sequence":"first","affiliation":[{"name":"Baylor College of Medicine, Houston, Texas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alan K.","family":"Percy","sequence":"additional","affiliation":[{"name":"University of Alabama at Birmingham, Birmingham, Alabama,"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathleen J.","family":"Motil","sequence":"additional","affiliation":[{"name":"Baylor College of 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