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
PTC Therapeutics
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; Neil Smith; APHENITY study group