Recent developments in the management of Huntington's disease

Huntington's disease (HD) is a rare, incurable, inherited neurodegenerative disorder manifested by chorea, hyperkinetic, and hypokinetic movements. The FDA has approved only two drugs, viz. tetrabenazine, and deu-tetrabenazine, to manage the chorea associated with HD. However, several other drugs are used as an off-label to manage chorea and other symptoms such as depression, anxiety, muscle tremors, and cognitive dysfunction associated with HD. So far, there is no disease-modifying treatment available. Drug repurposing has been a primary drive to search for new anti-HD drugs. Numerous molecular targets along with a wide range of small molecules and gene therapies are currently under clinical investigation. More than 200 clinical studies are underway for HD, 75% are interventional, and 25% are observational studies. The present review discusses the small molecule clinical pipeline and molecular targets for HD. Furthermore, the biomarkers, diagnostic tests, gene therapies, behavioral and observational studies for HD were also deliberated.

Automated summary

Huntington's disease is a rare and incurable neurodegenerative disorder. There are only two FDA-approved drugs for managing the symptoms associated with Huntington's disease, while other drugs are used off-label. Currently, drug repurposing and various clinical studies are being conducted to find new treatments for Huntington's disease.