Glycerol Phenylbutyrate Treatment in Children with Urea Cycle Disorders: Pooled Analysis of Short and Long-term Ammonia Control and Outcomes

OBJECTIVE: To evaluate glycerol phenylbutyrate (GPB) in the treatment of pediatric patients with urea cycle disorders (UCDs). STUDY DESIGN: UCD patients (n=26) ages 2 months through 17 years were treated with GPB and sodium phenylbutyrate (NaPBA) in two short-term, open-label crossover studies, whic...

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Detalhes bibliográficos
Publicado no:Mol Genet Metab
Main Authors: Berry, Susan A., Lichter-Konecki, Uta, Diaz, George A., McCandless, Shawn E., Rhead, William, Smith, Wendy, LeMons, Cynthia, Nagamani, Sandesh C.S., Coakley, Dion F., Mokhtarani, Masoud, Scharschmidt, Bruce F., Lee, Brendan
Formato: Artigo
Idioma:Inglês
Publicado em: 2014
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4382922/
https://ncbi.nlm.nih.gov/pubmed/24630270
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2014.02.007
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