OBJECTIVES: The completion of the genome project was expected to lead to treatments for genetically-linked disorders. In many cases this has not occurred. Our aim was to investigate the published research in one such disorder, Huntington’s disease (HD), in order to assess the current knowledge in this area. METHODS: A systematic search of Medline was conducted using a search filter for RCTs alongside keyword search strings for HD. Identified citations were first- and second-passed by two reviewers to determine inclusion/exclusion and reconciled by a third where decisions disagreed. Top-line data relating to treatment, included patients, and outcome was then extracted by two reviewers based on the abstract only. Differences were reconciled by a third reviewer. keyword search strings for HD. Identified citations were first- and second-passed by two reviewers to determine inclusion/exclusion and reconciled by a third where decisions disagreed. Top-line data relating to treatment, included patients, and outcome was then extracted by two reviewers based on the abstract only. Differences were reconciled by a third reviewer. RESULTS: A total of 397 probable RCTs were identified: 262 were excluded as non-RCTs and 135 included for extraction. Twenty-seven abstracts were unavailable, thus the final analysis is based on 108 studies. Of these studies, 29 were conducted in Europe, 29 in USA, 2 in Mexico, 2 in Israel, and one in Australia and Canada. The remaining studies did not state country of origin. Across all included studies 67 treatments were investigated, with between 3 and 537 patients enrolled. Almost all studies enrolled 30 individuals or less. Fifty-five studies reported that the intervention(s) was effective; 41 reported no efficacy (12 did not report the results). CONCLUSIONS: The majority of research in HD has been conducted in Western Europe and the United States. A wide variety of potential treatments have been investigated, many of which were ineffective for the treatment of HD. However, most research enrolled few patients and may thus lack power to detect any treatment effects. To date no genetically-related treatments have been developed, which is surprising given the identification of the HD gene in 1993. Further research into a cure for HD is warranted.