Glycerol phenylbutyrate efficacy and safety from an open label study in pediatric patients under 2?months of age with urea cycle disorders.
Longo N, Diaz GA, Lichter-Konecki U, Schulze A, Inbar-Feigenberg M, Conway RL, Bannick AA, McCandless SE, Zori R, Hainline B, Ah Mew N, Canavan C, Vescio T, Kok T, Porter MH, Berry SA. Glycerol phenylbutyrate efficacy and safety from an open label study in pediatric patients under 2?months of age with urea cycle disorders. Mol Genet Metab. 2021 01; 132(1):19-26.