Ammonia control in children with urea cycle disorders (UCDs); Phase 2 comparison of sodium phenylbutyrate and glycerol phenylbutyrate()

Twenty four hour ammonia profiles and correlates of drug effect were examined in a phase 2 comparison of sodium phenylbutyrate (NaPBA) and glycerol phenylbutyrate (GPB or HPN-100), an investigational drug being developed for urea cycle disorders (UCDs). STUDY DESIGN: Protocol HPN-100-005 involved op...

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Udgivet i:Mol Genet Metab
Main Authors: Lichter-Konecki, Uta, Diaz, G.A., Merritt, J.L., Feigenbaum, A., Jomphe, C., Marier, J.F., Beliveau, M., Mauney, J., Dickinson, K., Martinez, A., Mokhtarani, M., Scharschmidt, B., Rhead, W.
Format: Artigo
Sprog:Inglês
Udgivet: 2011
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4880058/
https://ncbi.nlm.nih.gov/pubmed/21612962
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2011.04.013
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