Abstract
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.
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Acknowledgements
The authors are grateful to their colleagues for their hard work and dedication to advocacy. We acknowledge the thoughtful contributions of L. Djurdjinovic, B. Conger and C. Driscoll. We thank Genetic Alliance for support for these advocacy organizations to increase their capacity. Most of all we thank the men, women and children who live with genetic conditions for their fortitude and passion for making a difference.
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Terry, S., Terry, P., Rauen, K. et al. Advocacy groups as research organizations: the PXE International example. Nat Rev Genet 8, 157–164 (2007). https://doi.org/10.1038/nrg1991
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DOI: https://doi.org/10.1038/nrg1991
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