Welcome to the seventeenth edition of the rare disease newsletter. A few months has changed the world of clinical research into a very different place. This issue includes a summary of work PRA has done to understand the impact of COVID19 on the rare disease patient community and to rare disease research.
Ask the Experts: What Does the Future Hold for CRISPR Gene Editing Technology?
Changing a living organism’s DNA sounds like something out of science fiction, but recent strides in genome (gene) editing have given scientists…
Rare Diseases Advisory Committee Members
The True Cost of Ineffective Clinical Trials
The process of designing and implementing clinical trials hasn’t changed much over the decades — which may be why 1 in 5 clinical trials fail due to…