New therapies for treating Down syndrome require quality of life measurement

Authors

  • Michael J. Goodman,

    Corresponding author
    1. Department of Pharmacotherapy, Pharmacotherapy Outcomes Research Center, University of Utah, Salt Lake City, Utah
    2. Down Syndrome Therapeutic Consortium, University of Utah, Salt Lake City, Utah
    • Assistant Professor, Pharmacotherapy Outcomes Research Center, 421 Wakara Way #208, Salt Lake City, UT 84108.
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  • Diana I. Brixner

    1. Department of Pharmacotherapy, Pharmacotherapy Outcomes Research Center, University of Utah, Salt Lake City, Utah
    2. Down Syndrome Therapeutic Consortium, University of Utah, Salt Lake City, Utah
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  • How to Cite this Article: Goodman MJ, Brixner DI. 2013. New therapies for treating Down syndrome require quality of life measurement. Am J Med Genet Part A 161A:639–641

  • The authors have no conflict of interest related to this publication.

Abstract

Down syndrome (DS) is the most common genetic cause of cognitive deficits. Using mouse models and therapies for Alzheimer disease, researchers are exploring therapies that may improve cognitive function in people with DS. These developments shift the health economic paradigm of understanding DS from determining the appropriate screening tool to the effect of therapy on quality of life in those with DS. To date, there are no validated quality of life instruments for DS. Research should begin to develop instruments that can evaluate changes in quality of life in therapeutic trials and beyond. © 2013 Wiley Periodicals, Inc.

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