Unmet Need
There is a need for a treatment that can slow down disease progression in ARG1-D
The symptoms and progressive manifestations of ARG1-D are challenging and burdensome for patients and their families1,2
The current standard of care for ARG1-D does not affect endogenousarginine production, and is thus not sufficient for sustainably normalisingplasma arginine levels over time3
The current guideline treatment goal for plasma arginine of 200 μmol/L is above the upper limit of normal and may not fully reverse ongoingmanifestations of ARG1-D4
*Normal range plasma arginine; 40-115 μmol/L, as defined in the PEACE trial
- Carvalho DR, et al. Clinical Features and Neurologic Progression of Hyperarginemia Pediatr Neurol. 2012;46:369-374.
- Jain-Ghai S, et al. Arginase I deficiency: Severe infantile presentation with hyperammonemia: More common than reported? Mol Genet Metab. 2011;104:107-111.
- Burrage LC, et al. Human recombinant arginase enzyme reduces plasma arginine in mouse models of arginase deficiency. Hum Mol Genet. 2015 Nov 15;24(22):6417-6427.
- Häberle J, et al. Suggested guidelines for the diagnosis and management of urea cycle disorders: First revision. J Inherit Metab Dis. 2019;1–39.