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

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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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Phenylketonuria Over Time: What We’re Learning from a Natural History Study

Rare Research Report: February 2024

Each month, we share summaries of recent Rare Diseases Clinical Research Network (RDCRN) grant-funded publications.

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National Survey Reveals Impact of COVID-19 on People Living with Rare Diseases and Their Families

The devastating impact of COVID-19 on the general population is well-documented—but less is known about the millions of people living with rare dis

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