PRA acknowledges and thanks the members of the Rare Disease Advisory
Committee (RDAC) who provided their valuable contributions to the
development of these guidelines.
Gene Therapy Research and Rare Diseases
RARE DISEASES NEWSLETTER Volume 7, October 2017
This quarterly publication will keep you up to date on PRA’s Rare Disease Team focus, achievements, and new initiatives.
How Technology is Reshaping the Rare Disease Landscape
This white paper provides practical strategies for easing the participation burden and introduces considerations for selecting technology that can…