| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 8343657 | Molecular Genetics and Metabolism | 2017 | 8 Pages |
Abstract
GPB was safe and effective in UCD patients aged 2Â months to 2Â years. GPB use was associated with good short- and long-term control of ammonia and glutamine levels, and the annualized frequency of hyperammonemic crises was lower during the study than before the study. There was no evidence for any previously unknown toxicity of GPB.
Keywords
OTCUCDCPS1PBAARG1ULNASsPagNN-acetylglutamate synthaseCDCPAAASLNAGSphenylacetategPbargininosuccinate lyaseBSATEAEarginase 1argininosuccinate synthetaseAmmoniaUrea cycle disorderUrea cycle disordersHACSodium benzoateOrnithine transcarbamylaseUpper limit of normalsodium phenylbutyratebody surface areabody mass indexBMITreatment-emergent adverse eventsPhenylbutyratePhenylacetylglutamineCenters for Disease Control and PreventionInfantsChildrenglutamineGlycerol phenylbutyrateNaPBA
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Authors
Susan A. Berry, Nicola Longo, George A. Diaz, Shawn E. McCandless, Wendy E. Smith, Cary O. Harding, Roberto Zori, Can Ficicioglu, Uta Lichter-Konecki, Beth Robinson, Jerry Vockley,