Rare Diseases Newsletter Volume 16, April 2020

COVID-19 Impact on Rare Disease Clinical Trials

Rare disease patient organizations are coping with a broad range of other organizational challenges, including reduced funding, canceled fundraising…

Gene Therapy Research and Rare Diseases

Guiding Principles for Interactions with Rare Disease Patient Advocacy Organizations

PRA acknowledges and thanks the members of the Rare Disease Advisory Committee (RDAC) who provided their valuable contributions to the development of…