Sepiapterin as a treatment for people living with phenylketonuria: a plain language summary of the APHENITY trial

20250 citationsJournal Articlediamond Open Access

Authors

Melissa Lah · Indiana University School of Medicine

Amaya Bélanger-Quintana · Hospital Universitario Ramón y Cajal

Nicola Longo · University of California, Los Angeles

Abstract

These results showed that sepiapterin helped to reduce blood Phe levels in the participants, and there were no unexpected safety concerns in people living with PKU. <b>ClinicalTrials.gov NCT number:</b> NCT05099640.

Topics & Keywords

Metabolism and Genetic Disorders Pharmacogenetics and Drug Metabolism Genomics and Rare Diseases

Publication Details

Published in: Journal of Comparative Effectiveness Research

Volume 15, Issue 1, pp. e250116-e250116

DOI: 10.57264/cer-2025-0116

Field-Weighted Citation Impact: 0.00

Command Palette

Sepiapterin as a treatment for people living with phenylketonuria: a plain language summary of the APHENITY trial

Authors

Abstract

Topics & Keywords

Publication Details