Research & Innovation | Overview
The Dr. Harvey Levy Program for Phenylketonuria and Related Conditions conducts research to advance our understanding of phenylketonuria (PKU) and homocystinuria (HCU), and the safety and efficacy of new treatments.
Clinical trials
Recruiting
- A Long Term, Post-Marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
- A Longitudinal Study of Hyperphenylalaninemia
Active (not recruiting)
- Non-invasive methods of assessing phenylalanine metabolism in individuals with phenylalanine hydroxylase (PAH) deficiency
- Pegtibatinase as an Enzyme Therapy for Patients With Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)
- Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
- A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
- Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU
- A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
- Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
Select publications
- The challenge of adults with phenylketonuria who have been lost to care; a single center's attempt to reach those diagnosed with PKU over 60 years of newborn screening
- Perspectives and Insights Into Phenylketonuria: Patient Narratives About the Early Years Following Newborn Screening
- Perspectives and Insights Into Phenylketonuria: Provider Narratives About the Early Years Following Newborn Screening
- Nutritional status of adults with phenylketonuria on pegvaliase: A 15-month prospective study
- Pegvaliase dosing in adults with PKU: Requisite dose for efficacy decreases over time
- Nutrition status of adults with phenylketonuria treated with pegvaliase
- First 1.5 years of pegvaliase clinic: Experiences and outcomes