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 MoreTraining 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

Consortium Spotlight: Advancing Discoveries in Phenylalanine Disorders
The Phenylalanine Families and Researchers Exploring Evide

Early Stage Investigator Spotlight: Stephanie Sacharow Helps Patients with Phenylketonuria Overcome Barriers to Treatment
Stephanie Sacharow, MD, is a medic
