Phase 2 Comparison of A Novel Ammonia Scavenging Agent With Sodium Phenylbutyrate In Patients With Urea Cycle Disorders: Safety, Pharmacokinetics And Ammonia Control

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 curre...

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Autors principals: Lee, Brendan, Rhead, William, Diaz, George A., Scharschmidt, Bruce F., Mian, Asad, Shchelochkov, Oleg, Marier, JF, Beliveau, Martin, Mauney, Joseph, Dickinson, Klara, Martinez, Antonia, Gargosky, Sharron, Mokhtarani, Masoud, Berry, Susan A.
Format: Artigo
Idioma:Inglês
Publicat: 2010
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC2905228/
https://ncbi.nlm.nih.gov/pubmed/20382058
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2010.03.014
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