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Nature Reviews Genetics 8, 157–164 (1 February 2007) | doi:10.1038/nrg1991

Advocacy groups as research organizations: the PXE International example

Sharon F. Terry , Patrick F. Terry , Katherine A. Rauen , Jouni Uitto & Lionel G. Bercovitch

Advocacy organizations for genetic diseases are increasingly becoming involved in biomedical research, particularly translational research, in order to meet the needs of the individuals that they serve. PXE International, an advocacy organization for the disease pseudoxanthoma elasticum, provides an example of how research can be accelerated by these groups. It has adopted methods that were pioneered by other advocacy organizations, and has integrated these along with new approaches into franchizable elements. The model has been followed for other conditions and has led to the establishment of a common infrastructure to enable advocacy groups to initiate, conduct and accelerate research.