RARE DISEASES NEWSLETTER Volume 6, May 2017

Decoding FDA Gene Therapy Guidances Part Two: Rare Disease

In Part Two of Decoding FDA Gene Therapy Guidances, we discuss three separate guidances that provide further insight into how sponsors can safely and…

Rare Diseases and the Promise of Gene Therapy: What You Need to Know

Rare Diseases Advisory Committee

Rare Diseases Advisory Committee—PRA is proud to join forces with a group of highly skilled and experienced patient advocates — collectively working…