Pharmacogenetics in the Treatment of Huntington's Disease: Review and Future Perspectives.

ABSTRACT: Huntington's disease (HD) is an autosomal dominant progressive brain disorder, caused by a pathological expansion of a CAG repeat that encodes the huntingtin gene. This genetic neurodegenerative rare disease is characterized by cognitive, motor, and neuropsychiatric manifestations. The aim of the treatment is symptomatic and addresses the hyperkinetic disorders (chorea, dystonia, myoclonus, tics, etc.) and the behavioural and cognitive disturbances (depression, anxiety, psychosis, etc.) associated with the disease. HD is still a complex condition in need of innovative and efficient treatment. The long-term goal of pharmacogenetic studies is to use genotype data to predict the effective treatment response to a specific drug and, in turn, prevent potential undesirable effects of its administ

SUBMITTER: Garcia-Gonzalez X 

PROVIDER: S-EPMC10056055 | biostudies-literature | 2023 Feb

REPOSITORIES: biostudies-literature