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Phase 2 comparison of a novel ammonia scavenging agent with sodium phenylbutyrate in patients with urea cycle disorders: safety, pharmacokinetics and ammonia control.

Mol Genet Metab 2010 Jul 23;100(3):221-8. Epub 2010 Mar 23.

Baylor College of Medicine, Houston, TX, USA.

Unlabelled: Glycerol phenylbutyrate (glyceryl tri (4-phenylbutyrate)) (GPB) is being studied as an alternative to sodium phenylbutyrate (NaPBA) for the treatment of urea cycle disorders (UCDs). This phase 2 study explored the hypothesis that GPB offers similar safety and ammonia control as NaPBA, which is currently approved as adjunctive therapy in the chronic management of UCDs, and examined correlates of 24-h blood ammonia.

Methods: An open-label, fixed sequence switch-over study was conducted in adult UCD patients taking maintenance NaPBA. Read More

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