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Updated: May 26, 2026

Fractionation for Resolution of Soluble and Insoluble Huntingtin Species
Published on: February 27, 2018
An evidence-based approach in the treatment of Huntington's disease
1Neurological Clinical Research Unit, Instituto de Medicina Molecular, Lisbon, Portugal. tmestre@gmail.com
Insights
Huntington's disease treatments lack strong evidence, with only tetrabenazine approved for chorea. Ongoing research offers hope for future Huntington's disease management strategies.
Area of Science:
- Neuroscience
- Neurology
- Clinical Pharmacology
Background:
- Huntington's disease (HD) is a progressive neurodegenerative disorder with varied symptoms.
- Currently, no cure or disease-modifying treatments exist for HD.
- Tetrabenazine is the sole FDA-approved drug, specifically for chorea management.
Purpose of the Study:
- To review updated clinical trial results and ongoing research for effective Huntington's disease treatments.
- To assess therapeutic options for motor, neuropsychiatric, and cognitive symptoms of HD.
Main Methods:
- Systematic review of well-designed randomized controlled trials.
- Analysis of recent clinical trial data and ongoing research efforts.
Main Results:
- Limited evidence supports most current treatment decisions for Huntington's disease.
- Expert opinion-based recommendations currently guide clinical practice.
Conclusions:
- Most Huntington's disease treatments lack robust supporting evidence.
- Clinical management relies heavily on expert consensus.
- Significant ongoing research is anticipated to advance HD treatment in the future.
Abstract:
Huntington's disease (HD) is a neurodegenerative disease with diverse symptoms for which there is no curative or disease-modifying treatment available. Currently, tetrabenazine is the only drug approved for HD by a regulatory agency, and only for the treatment of chorea. In the current review, we present updated results from recent clinical trials and ongoing clinical research efforts to find effective and safe treatments for HD motor, and neuropsychiatric and cognitive symptoms. We used a systematic review approach that included data from well-designed randomised controlled trials. The authors conclude that there is weak evidence to support most of the treatment decisions in HD and thus clinicians may be guided only by expert opinion-based therapeutic recommendations. Ongoing research is considerable and is expected to have an impact in the management of HD in upcoming years.
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