Waisman Center
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Long-term safety and efficacy of glycerol phenylbutyrate for the management of urea cycle disorder patients

Rhead, MD, PHD, W. Long-Term Safety and Efficacy of Glycerol Phenylbutyrate for the Management of Urea Cycle Disorder Patients.

Diaz GA, Schulze A, Longo N, Rhead W, Feigenbaum A, Wong D, Merritt JL 2nd, Berquist W, Gallagher RC, Bartholomew D, McCandless SE, Smith WE, Harding CO, Zori R, Lichter-Konecki U, Vockley J, Canavan C, Vescio T, Holt RJ, Berry SA. (2019). Long-term safety and efficacy of glycerol phenylbutyrate for the management of urea cycle disorder patients. Molecular Genetics and Metabolism, 127(4):336-345. doi: 10.1016/j.ymgme.2019.07.004.