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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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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

Consortium Spotlight: Advancing Discoveries in Phenylalanine Disorders

The Phenylalanine Families and Researchers Exploring Evide

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Headshot of Stephanie Sacharow, MD