Potter, Beth K.
Khangura, Sara D.
Tingley, Kylie
Chakraborty, Pranesh
Little, Julian
This article is maintained by: Elsevier
Article Title: Translating rare-disease therapies into improved care for patients and families: what are the right outcomes, designs, and engagement approaches in health-systems research?
Journal Title: Genetics in Medicine
CrossRef DOI link to publisher maintained version: https://doi.org/10.1038/gim.2015.42
Content Type: article
Copyright: © 2016, The Author(s), under exclusive licence to the American College of Medical Genetics and Genomics