PKU Program | Research & Innovation

Research & Innovation

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

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)

Dr. Harvey Levy Program for Phenylketonuria and Related Conditions

Research & Innovation

Clinical trials

Recruiting

Active (not recruiting)

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