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TITLE: 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  Full Text
AUTHORS: Ania Muntau; Nicola Longo; Fatih Ezgu; Ida Vanessa 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; Neil Smith; 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; ...More
PUBLISHED: 2024, SOURCE: LANCET, VOLUME: 404, ISSUE: 10460
INDEXED IN: Scopus WOS
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TITLE: Parkinson's disease in GTP cyclohydrolase 1 mutation carriers  Full Text
AUTHORS: Niccolo E Mencacci; Ioannis U Isaias; Martin M Reich; Christos Ganos; Vincent Plagnol; James M Polke; Jose Bras; Joshua Hersheson; Maria Stamelou; Alan M Pittman; Alastair J Noyce; Kin Y Mok; Thomas Opladen; Erdmute Kunstmann; Sybille Hodecker; Alexander Muenchau; Jens Volkmann; Samuel Samnick; Katie Sidle; Tina Nanji; Mary G Sweeney; Henry Houlden; Amit Batla; Anna L Zecchinelli; Gianni Pezzoli; Giorgio Marotta; Andrew Lees; Paulo Alegria; Paul Krack; Florence Cormier Dequaire; Suzanne Lesage; Alexis Brice; Peter Heutink; Thomas Gasser; Steven J Lubbe; Huw R Morris; Pille Taba; Sulev Koks; Elisa Majounie; Raphael Gibbs; Andrew Singleton; John Hardy; Stephan Klebe; Kailash P Bhatia; Nicholas W Wood; ...More
PUBLISHED: 2014, SOURCE: BRAIN, VOLUME: 137, ISSUE: 9
INDEXED IN: Scopus WOS CrossRef: 72