A randomized controlled trial to evaluate the effects of high-dose versus low-dose of arginine therapy on hepatic function tests in argininosuccinic aciduria

SCS Nagamani, OA Shchelochkov, MA Mullins… - Molecular genetics and …, 2012 - Elsevier
OBJECTIVE: To compare the effects of combinatorial therapy with low-dose arginine and a
nitrogen scavenging agent (sodium phenylbutyrate) vs. monotherapy with high-dose
arginine on liver function tests in patients with argininosuccinic aciduria (ASA). STUDY
DESIGN: Twelve patients with ASA were enrolled in a double-blind, placebo-controlled,
cross-over study design. Subjects were randomized to receive either a low-dose of arginine
therapy (100mg· kg− 1· d− 1) combined with sodium phenylbutyrate (500mg· kg− 1· d …