New therapies for treating Down syndrome require quality of life measurement.

Authors :: Goodman MJ
Brixner DI
Source :: American journal of medical genetics. Part A [Am J Med Genet A] 2013 Apr; Vol. 161A (4), pp. 639-41. Date of Electronic Publication: 2013 Mar 12.
Publication Year :: 2013
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.<br /> (Copyright © 2013 Wiley Periodicals, Inc.)