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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.

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Join a Research Study

Join the RDCRN for Rare Disease Day at NIH on February 27, 2026

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

Rare Disease Day at NIH. February 27, 2026. #RDDNIH. ncats.nih.gov/rdd

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

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PHEFREE Updates

Newsletter of the Phenylalanine Families and Researchers Exploring Evidence

PHEFREE January 2026 Consortium Updates

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