Rare disease patient organizations are coping with a broad range of other organizational challenges, including reduced funding, canceled fundraising events, sharp increases in requests for information and support from the patients they serve, and navigating the shift to remote work.
RARE DISEASES NEWSLETTER Volume 8, January 2018
This quarterly publication will keep you up to date on PRA’s Rare Disease Team focus, achievements, and new initiatives.
Decoding FDA Gene Therapy Guidances Part One: Chemistry, Manufacturing, and Control
In Part One of our Decoding FDA Gene Therapy Guidances series, we discuss the significance of several new FDA guidances regarding human gene therapy…
Partnering with Patients to Develop Meaningful Endpoints – Practical Considerations
Selection of appropriate endpoints for rare disease clinical studies can be challenging but is critical to assessing the efficacy of new medicines.…