The hunt for better treatments for Huntington's disease.
Mason, Sarah L
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Barker, R., & Mason, S. L. (2019). The hunt for better treatments for Huntington's disease.. Lancet Neurol, 18 (2), 131-133. https://doi.org/10.1016/S1474-4422(18)30448-4
Huntington’s Disease (HD) is an autosomal dominant condition that typically presents in midlife with a combination of motor, cognitive and psychiatric problems along with sleep and metabolic abnormalities. It runs a clinical course over 15-20 years leading to death as patients become demented and bed bound. There are currently no proven disease modifying therapies for HD. While the recent work with anti-sense therapies (ASO) against huntingtin has generated much excitement they are yet to demonstrate a measurable change in disease progression. Furthermore, given they promise to “modify” and not “cure” HD patients we will still require adjunct symptomatic treatments, potentially for longer periods of time. Symptomatic therapies for HD do exist and are widely used for treating the chorea and some of the psychiatric aspects of the condition although the extent to which they work is variable1 and many experience significant side effects with them. Therefore, there is still a need to develop better symptomatic therapies, ideally with single drugs that can help treat more than one sign and symptom of this condition.
Humans, Huntington Disease, Piperidines
Cambridge University Hospitals NHS Foundation Trust (CUH) (146281)
Huntington's Disease Association of England and Wales (HD Economics)
External DOI: https://doi.org/10.1016/S1474-4422(18)30448-4
This record's URL: https://www.repository.cam.ac.uk/handle/1810/287598
Attribution-NonCommercial-NoDerivatives 4.0 International
Licence URL: https://creativecommons.org/licenses/by-nc-nd/4.0/