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An update on Huntington's disease: from the gene to the clinic.
Kim, Samuel D; Fung, Victor S C.
Affiliation
  • Kim SD; aMovement Disorders Unit, Department of Neurology, Westmead Hospital bSydney Medical School-Western, University of Sydney, Sydney, Australia.
Curr Opin Neurol ; 27(4): 477-83, 2014 Aug.
Article in En | MEDLINE | ID: mdl-24978638
PURPOSE OF REVIEW: This review highlights the recent advances in Huntington's disease, with a particular focus on development of disease biomarkers for use in therapeutic trials in the premotor phase of the disease, as well as the growing literature regarding pathophysiological mechanisms and their relevance to potential therapeutic targets. RECENT FINDINGS: There have been continued advances in the development of disease biomarkers, and promising neuroprotection trials are beginning to emerge in the premotor stage of Huntington's disease. Deeper understanding of the pathophysiological mechanisms is being translated into potential therapeutic strategies. SUMMARY: The premotor stage of Huntington's disease provides an ideal time to trial disease-modifying therapy, but reliable biomarkers are required for monitoring disease progression, and this remains an area of intense research. Our understanding of the underlying pathophysiological mechanisms continues to expand, and a number of promising therapeutic strategies are emerging, including strategies to silence mutant huntingtin expression.
Subject(s)

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Huntington Disease Limits: Humans Language: En Journal: Curr Opin Neurol Journal subject: NEUROLOGIA Year: 2014 Document type: Article Affiliation country: Australia Country of publication: United kingdom

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Huntington Disease Limits: Humans Language: En Journal: Curr Opin Neurol Journal subject: NEUROLOGIA Year: 2014 Document type: Article Affiliation country: Australia Country of publication: United kingdom