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.
Learn MoreRare Research Report: February 2024
Each month, we share summaries of recent Rare Diseases Clinical Research Network (RDCRN) grant-funded publications.
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