Improving the lives of those with phenylketonuria (PKU) through clinical research.
Find a Clinical Site Near You
Participants make it possible for researchers to find new treatments, speed diagnosis and improve the lives of those affected by rare diseases.
Learn More
NIH Announces Funding to Establish and Strengthen Rare Disease Research Groups
The National Institutes of Health (NIH) has awarded approximately $26 million in grants in the fiscal year 2025 to begin the fifth cycle of funding
$27M Grant Renews Cincinnati Children’s as Coordinating Center for the Rare Diseases Clinical Research Network
The federal agency that oversees a large network of research centers seeking cures for hundreds of rare, disabling, and often fatal diseases has re
Pre-Doc Preparatory Program (P³) in Translational Research
Do you have a rare disease research coordinator, genetic counseling assistant, or technician who may benefit from formal training in clinical and t
