Effects of oral sepiapterin on blood Phe concentration in a broad range of patients with phenylketonuria (APHENITY): results of an international, phase 3, randomised, double-blind, placebo-controlled trial

作者：Ania Carolina Muntau, Ania C Muntau, Nicola Longo, Fatih Süheyl Ezgü, Ida Vanessa Döederlein Schwartz, Melissa D. Lah, Drago Bratkovic, Lali Margvelashvili, Ertuğrul Kıykım, Roberto T. Zori, Jaume Campistol Plana, Amaya Bélanger-Quintana, Allan Meldgaard Lund, Laura Guilder, Anupam Chakrapani, Halise Neslihan Mungan, Arlindo Guimas, Ixiu del Carmen Cabrales Guerra, Anita MacDonald, Kimberly A. Ingalls, Neil Smith, Ania C Muntau, Ania C Muntau, Nicola Longo, Fatih Ezgu, Ida Vanessa D Schwartz, Melissa Lah, Drago Bratkovic, Lali Margvelashvili, Ertugrul Kiykim, Roberto Zori, Jaume Campistol Plana, Amaya Bélanger-Quintana, Allan Lund, Laura Guilder, Anupam Chakrapani, Halise Neslihan Mungan, Arlindo Guimas, Ixiu del Carmen Cabrales Guerra, Anita MacDonald, Kimberly Ingalls, Stephanie Sacharow, Maria Avanise Yumi Minami, Frank Rutsch, Michel Tchan, Heidi Peters, Aneal Khan, Suresh Vijay, Thomas Opladen, Hope Northrup, Anabela Oliveira, François Feillet, Dodo Agladze, Catalina Hughes, Alexandra Larkin, Sarah Milner, Lan Gao, Rochelle Greenbaum, Zhenming Zhao, Neil Smith · 发表于：The Lancet · 年份：2024 · DOI：10.1016/s0140-6736(24)01556-3 · 被引用次数：43 · 研究领域：Metabolism and Genetic Disorders、Pharmacogenetics and Drug Metabolism、Genomics and Rare Diseases