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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...
Wedi'i Gadw mewn:
Cyhoeddwyd yn: | Mol Genet Metab |
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Prif Awduron: | , , , , , , , , , , , |
Fformat: | Artigo |
Iaith: | Inglês |
Cyhoeddwyd: |
2014
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Pynciau: | |
Mynediad Ar-lein: | 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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