Therapeutic options for Huntington's disease

Curr Opin Investig Drugs. 2003 Jan;4(1):51-4.

Abstract

Huntington's disease (HD) is an autosomal dominant, progressive neurodegenerative disorder. The genetic defect lies in the expansion of a CAG repeat on chromosome 4 and neuropathologically it is characterized by neuronal loss in the striatum. Clinical signs are chorea, impaired voluntary movement, behavioral changes and dementia. Present therapies are limited to these symptoms without any influence on the course of the disease, whereas current pharmacological developments mainly focus on delaying disease progression. This review discusses the present symptomatic treatments and focuses on recent developments of new therapeutic approaches for HD.

Publication types

  • Review

MeSH terms

  • Animals
  • Cell Transplantation
  • Clinical Trials as Topic
  • Corpus Striatum / cytology
  • Humans
  • Huntington Disease / drug therapy
  • Huntington Disease / genetics
  • Huntington Disease / therapy*