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
安慰剂
双盲
医学
内科学
替代医学
病理
作者
Ania C. Muntau,Nicola Longo,Fatih Süheyl Ezgü,Ida Vanessa Döederlein Schwartz,Melissa Lah,Drago Bratkovic,Lali Margvelashvili,Ertuğrul Kıykım,David Kronn,Jaume Campistol Plana,Amaya Bélanger-Quintana,Allan M. Lund,Laura Guilder,Anupam Chakrapani,Neslihan Önenli Mungan,Arlindo Guimas,Ixiu del Carmen Cabrales Guerra,Anita MacDonald,Kimberly Ingalls,Neil Smith
Phenylketonuria is an inherited condition characterised by neurotoxic accumulation of phenylalanine (Phe). APHENITY assessed the efficacy and safety of orally administered synthetic sepiapterin in children and adults with phenylketonuria.
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Jasper
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Singularity
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