Journal article
Huntington disease: natural history, biomarkers and prospects for therapeutics
Nature reviews. Neurology, Vol.10(4), pp.204-216
04/2014
DOI: 10.1038/nrneurol.2014.24
PMID: 24614516
Abstract
Huntington disease (HD) can be seen as a model neurodegenerative disorder, in that it is caused by a single genetic mutation and is amenable to predictive genetic testing, with estimation of years to predicted onset, enabling the entire range of disease natural history to be studied. Structural neuroimaging biomarkers show that progressive regional brain atrophy begins many years before the emergence of diagnosable signs and symptoms of HD, and continues steadily during the symptomatic or 'manifest' period. The continued development of functional, neurochemical and other biomarkers raises hopes that these biomarkers might be useful for future trials of disease-modifying therapeutics to delay the onset and slow the progression of HD. Such advances could herald a new era of personalized preventive therapeutics. We describe the natural history of HD, including the timing of emergence of motor, cognitive and emotional impairments, and the techniques that are used to assess these features. Building on this information, we review recent progress in the development of biomarkers for HD, and potential future roles of these biomarkers in clinical trials.
Details
- Title: Subtitle
- Huntington disease: natural history, biomarkers and prospects for therapeutics
- Creators
- Christopher A Ross - Division of Neurobiology, Johns Hopkins University, 600 North Wolfe Street, Baltimore, MD 21287, USAElizabeth H Aylward - Seattle Children's Research Institute, USAEdward J Wild - University College London, UKDouglas R Langbehn - University of Iowa, USAJeffrey D Long - University of Iowa, USAJohn H Warner - CHDI Management/CHDI Foundation, USARachael I Scahill - University College London, UKBlair R Leavitt - University of British Columbia, CanadaJulie C Stout - Monash University, AustraliaJane S Paulsen - University of Iowa, USARalf Reilmann - George-Huntington-Institute, GermanyPaul G Unschuld - University of Zürich, SwitzerlandAlice Wexler - University of California, Los Angeles, USARussell L Margolis - Division of Neurobiology, Johns Hopkins University, 600 North Wolfe Street, Baltimore, MD 21287, USASarah J Tabrizi - University College London, UK
- Resource Type
- Journal article
- Publication Details
- Nature reviews. Neurology, Vol.10(4), pp.204-216
- DOI
- 10.1038/nrneurol.2014.24
- PMID
- 24614516
- NLM abbreviation
- Nat Rev Neurol
- ISSN
- 1759-4766
- eISSN
- 1759-4766
- Publisher
- England
- Grant note
- P30 HD002274 / NICHD NIH HHS R01 NS040068 / NINDS NIH HHS
- Language
- English
- Date published
- 04/2014
- Academic Unit
- Psychiatry; Psychological and Brain Sciences; Iowa Neuroscience Institute; Biostatistics
- Record Identifier
- 9984004182402771
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