Home | Metabolism | Phenylketonuria (PKU) | Publications | Publication: PTC923 (sepiapterin) lowers elevated blood phenylalanine in subjects with phenylketonuria: A Phase 2 randomized, multi-center, three-period crossover, open-label, active controlled, all-comers study

Publication: PTC923 (sepiapterin) lowers elevated blood phenylalanine in subjects with phenylketonuria: A Phase 2 randomized, multi-center, three-period crossover, open-label, active controlled, all-comers study

Published in Metabolism Clinical and Experimental, this research article presents the real-world efficacy of PTC923, compared with sapropterin, in a Phase 2 study of patients with phenylketonuria (PKU)

  • Understand the effect of PTC923 on blood phenylalanine (Phe) levels in patients with PKU
  • Find out about the relationship between different doses of PTC923 and blood Phe levels
  • Review the safety and efficacy of PTC923 in this study

Bratkovic D, Margvelashvili L, Tchan MC, et al. Metabolism 2022;128:155116

Sepiapterin is an investigational drug currently in development for PKU.

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US-PKU-0208 | April 2023
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