Get 20M+ Full-Text Papers For Less Than $1.50/day. Start a 14-Day Trial for You or Your Team.

Learn More →

Will genotype drive treatment options?

Will genotype drive treatment options? Individual genetic variation can have a major impact on the clinical manifestation of a movement disorder and its response to treatment. Advances in gene discovery and increasing availability of diagnostic genetic testing have led to the identification of a growing number of patients with well‐defined hereditary movement disorders. Establishing a genetic diagnosis may greatly impact patient counseling and shape therapeutic decisions. Further, assignment of a movement disorder to a specific genetic defect holds promise for the development of causal treatment approaches and individualized therapies, especially as the first gene‐targeted approaches have recently entered clinical trials. However, important gaps remain, that is, genetic testing results are often inconclusive, gene‐specific treatment options are still exceedingly rare, and designing clinical trials to demonstrate disease modification continues to pose a major challenge. © 2019 International Parkinson and Movement Disorder Society http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Movement Disorders Wiley

Will genotype drive treatment options?

Brüggemann, Norbert; Klein, Christine
Movement Disorders , Volume 34 (9) – Sep 1, 2019
Download PDF
6 pages

Loading next page...
 
/lp/wiley/will-genotype-drive-treatment-options-4pLZWFxVUK

References (27)

Publisher
Wiley
Copyright
© 2019 International Parkinson and Movement Disorder Society
ISSN
0885-3185
eISSN
1531-8257
DOI
10.1002/mds.27699
Publisher site
See Article on Publisher Site

Abstract

Individual genetic variation can have a major impact on the clinical manifestation of a movement disorder and its response to treatment. Advances in gene discovery and increasing availability of diagnostic genetic testing have led to the identification of a growing number of patients with well‐defined hereditary movement disorders. Establishing a genetic diagnosis may greatly impact patient counseling and shape therapeutic decisions. Further, assignment of a movement disorder to a specific genetic defect holds promise for the development of causal treatment approaches and individualized therapies, especially as the first gene‐targeted approaches have recently entered clinical trials. However, important gaps remain, that is, genetic testing results are often inconclusive, gene‐specific treatment options are still exceedingly rare, and designing clinical trials to demonstrate disease modification continues to pose a major challenge. © 2019 International Parkinson and Movement Disorder Society

Journal

Movement Disorders – Wiley

Published: Sep 1, 2019

Keywords: ; ; ; ;

There are no references for this article.