Glycerol phenylbutyrate efficacy and safety from an open label study in pediatric patients under 2 months of age with urea cycle disorders.

Autor: Longo, Nicola1 (AUTHOR), Diaz, George A.2 (AUTHOR), Lichter-Konecki, Uta3 (AUTHOR), Schulze, Andreas4 (AUTHOR), Inbar-Feigenberg, Michal4 (AUTHOR), Conway, Robert L.5 (AUTHOR), Bannick, Allison A.5 (AUTHOR), McCandless, Shawn E.6 (AUTHOR), Zori, Roberto7 (AUTHOR), Hainline, Bryan8 (AUTHOR), Ah Mew, Nicholas9 (AUTHOR), Canavan, Colleen10 (AUTHOR), Vescio, Thomas10 (AUTHOR), Kok, Teresa1,10 (AUTHOR) Tkok@horizontherapeutics.com, Porter, Marty H.10 (AUTHOR), Berry, Susan A.11 (AUTHOR)
Zdroj: Molecular Genetics & Metabolism. Jan2021, Vol. 132 Issue 1, p19-26. 8p.
Databáze: Academic Search Ultimate
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