Rare Disease

Three Necessary Steps When Designing your New World Protocol

This article explores the design of a protocol that includes decentralized components. After a clinical-trial protocol has been developed, adding…

RARE DISEASES NEWSLETTER Volume 17, July 2020

Welcome to the seventeenth edition of the rare disease newsletter. A few months has changed the world of clinical research into a very different…

Industry Watch

This week we bring you news of a gene therapy with the potential to change lives for children with Duchenne muscular dystrophy, having experienced…

Industry Watch

This week’s issue of Industry Watch is focused on proposed changes to US lung cancer screening guidelines which will hopefully result in increased…

Rare Disease Newsletter

This quarterly publication will keep you informed about key news in the world of rare disease clinical research and update you on what the PRA Health…

Center for Rare Diseases continues to engage with rare disease patient communities during COVID-19 crisis

PRA Health Sciences’ Center for Rare Diseases is committed to engaging rare disease patient communities, and providing transparency, throughout the…

Creating Solutions to Ensure Endpoint Protection in Challenging Rare Disease Trial

The collection of high-quality endpoint data was paramount to the success of this Phase II program. Because this was the first clinical trial ever…

PRA Talks COVID-19 Impact on Rare Disease Clinical Trials with Patient Advocates

PRA’s Center for Rare Diseases understands that clinical trials are both a source of hope and care for people affected by a rare disease. The impact…

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…

Rare Diseases Newsletter Volume 16, April 2020