Introduction: Huntington's disease (HD) is an incurable chronic neurodegenerative disorder that typically presents in mid-life with a range of motor, cognitive and affective problems. Patients are currently managed using a combination of drug treatments and non-pharmacological therapies but at present there is no "gold standard" treatment for any aspect of the disease.
Areas covered: In this review the empirical evidence supporting the use of drugs commonly used to treat HD was discussed. In particular, we focus on therapeutics that have either reached phase 3 clinical trials or are already in clinical use.
Expert opinion: The results confirmed that there is a striking lack of evidence to support the efficacy of the drugs currently used in the management of HD. In fact, many drugs are prescribed on the basis of case reports, open label studies, small double blind placebo control trials of limited duration, or personal clinical experience. However of late, the establishment of large international databases, capturing all patients and their clinical details regardless of stage or geographical location has led to an increase in the number of clinical trials conducted on new therapies. Unfortunately, the same is not true for the existing therapies which look set to remain untested for the foreseeable future.
Keywords: ENROLL-HD; Huntington’s disease; drug trials; evidence-based practice; symptomatic therapies.