Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: Interim results from the phase 3 APHENITY Extension Study

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Publication Details

Journal
Genetics in Medicine
Published
2026-01-12
DOI
https://doi.org/10.1016/j.gim.2026.101683
Citations
1
Primary Topic
Metabolism and Genetic Disorders
Type
article
Field-Weighted Citation Impact
41.00
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article

Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: Interim results from the phase 3 APHENITY Extension Study

Maria Avanise Yumi Minami, Kimberly Ingalls, Ebru Canda, Alex Larkin et al.
1 citations
Genetics in Medicine
Metabolism and Genetic Disorders
41.00
article

Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: Interim results from the phase 3 APHENITY Extension Study

Maria Avanise Yumi Minami, Kimberly Ingalls, Ebru Canda, Alex Larkin, Olivia Fjellbirkeland, Dodo Agladze, Filippo Manti, Mika Ishige, Neslihan Önenli Mungan, Enwu Liu, Margo Sheck Breilyn, Melissa Lah, Takashi Hamazaki, Patrícia Janeiro, Frank Rutsch, Opladen Thomas, Nicola Longo, Aneal Khan, Ida Vanessa D. Schwartz, Anita MacDonald, Ertuğrul Kiykim, Roberto Zori, Anita Inwood, Yılmaz Yıldız, Heidi Peters, Jerry Vockley, Stephanie Sacharow, Jaume Campistol Plana, Janet A. Thomas, Anupam Chakrapani, Lan Gao, Fatih Ezgü, Catalina Hughes, Lali Margvelashvili, Ixiu-Cabrales Guerra, Tetsuya Ito, Ania C. Muntau, Francjan van Spronsen, Allan Lund, Anabela Oliveira, Neil Smith, Alberto Burlina, Laura Guilder, Arlindo Guimas, Michel Tchan, Amaya Bélanger-Quintana, Suresh Vijay
article en
1 citations

Abstract

No abstract available for this paper.
Genetics in MedicineVol. 28(4)
Nihon University (JP), Boston Children's Hospital (US), Hospital Sant Joan de Déu Barcelona (ES), Royal Children's Hospital (AU), University Medical Center Groningen (NL), University of Padua (IT), Fujita Health University (JP), Universität Hamburg (DE), University of California, Los Angeles (US), University of Pittsburgh (US), Heidelberg University (DE), Great Ormond Street Hospital (GB), Women's and Children's Hospital (AU), Hospital for Sick Children (CA), University Hospital Heidelberg (DE), Copenhagen University Hospital (DK), Rigshospitalet (DK), Westmead Hospital (AU), University of Florida (US), Ege University (TR), Hospital de Clínicas de Porto Alegre (BR), Birmingham Children's Hospital (GB), University Hospital Münster (DE), Center for Human Genetics (US), University Medical Center Hamburg-Eppendorf (DE), Kinderkrebs-Zentrum Hamburg (DE), Istanbul University-Cerrahpaşa (TR), Centro Hospitalar Lisboa Norte (PT), Pittsburgh Public Schools (US), AIDS and Clinical Immunology Research Center (GE), Institute of Human Genetics (PL), PTC Therapeutics (United States) (US), National Cancer Center of Georgia (GE), Hospital de Santa Maria (PT), Centro Hospitalar do Porto (PT), Clinics Hospital of Ribeirão Preto (BR), Magna International (Canada) (CA), Hospital Universitario Ramón y Cajal (ES), Children's Health Queensland Hospital and Health Service (AU), Indiana University School of Medicine, University College London (GB), Indiana University – Purdue University Indianapolis (US), Cukurova University (TR), Hacettepe University (TR), Tokyo Metropolitan University (JP), Pomeranian Medical University (PL), Sapienza University of Rome (IT), Universidad Panamericana (MX), University of Colorado Denver (US), Florida College (US), Gazi University (TR), Icahn School of Medicine at Mount Sinai (US)
Zero hunger
Openalex Percentile: Top 2%
Metabolism and Genetic Disorders
41.00
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