Safety and efficacy of glycerol phenylbutyrate for management of urea cycle disorders in patients aged 2Â months to 2Â years

GPB was safe and effective in UCD patients aged 2 months to 2 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.