Sepiapterin as a treatment for people living with phenylketonuria: a plain language summary of the APHENITY trial
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.
Authors
- Ania C. Muntau (ORCID: https://orcid.org/0000-0002-2900-8378)
- Amaya Bélanger-Quintana (ORCID: https://orcid.org/0000-0003-0102-8956)
- Melissa Lah (ORCID: https://orcid.org/0000-0002-2406-8961)
- Sarah Gallagher (ORCID: https://orcid.org/0000-0003-3515-7035)
- Nicola Longo (ORCID: https://orcid.org/0000-0002-3677-1216)
Institutions
- Universität Hamburg (DE)
- University of California, Los Angeles (US)
- German Center for Pediatric and Adolescent Rheumatology (DE)
- University Medical Center Hamburg-Eppendorf (DE)
- National PKU Alliance (US)
- Hospital Universitario Ramón y Cajal (ES)
- Indiana University School of Medicine
- Indiana University – Purdue University Indianapolis (US)
Publication Details
- Journal
- Journal of Comparative Effectiveness Research
- Published
- 2025-12-09
- DOI
- https://doi.org/10.57264/cer-2025-0116
- Primary Topic
- Metabolism and Genetic Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Sanofi
- PTC Therapeutics
- Moderna
- Ipsen