Skip to main content

Improving the lives of those with phenylketonuria (PKU) through clinical research.

A man and a woman each have a child sitting on their lap.

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
Map pinpoint icon

Join a Research Study

Join RDCRN at Rare Disease Day at NIH on February 28

Don’t miss the in-person and virtual celebration of

Rare Disease Day at NIH. February 28, 2023. #RDDNIH.

Training Program for Clinical Research in Rare Disease Launches New Session

The National Institutes of Health-funded R25 Rare Disease Clinical Research Training Program is launching a new training program for clinical resea

Screenshot from a presentation on the Rare Diseases Research R25 Curriculum Grant
Phenylalanine Families and Researchers Exploring Evidence (PHEFREE) logo