Russo RS, Gasperini S, Bubb G, Neuman L, Sloan LS et al; PEACE Investigators.
Efficacy and safety of pegzilarginase in arginase 1 deficiency (PEACE): a phase 3, randomized, double-blind, placebo-controlled, multi-centre trial.
EClinicalMedicine. 2024 ;68:102405
Soria LR, Ah Mew N, Brunetti-Pierri N.
Progress and challenges in development of new therapies for urea cycle disorders.
Hum Mol Genet. 2019 ;28(R1):R42-R48
Duff C, Alexander IE, Baruteau J.
Gene therapy for urea cycle defects: An update from historical perspectives to future prospects.
J Inherit Metab Dis. 2024 ;47:50-62