Arginase 1 deficiency (ARG1-D) research hub

A Phase 3 Open-Label Study of Safety, Pharmacokinetics and Activity of Weekly Subcutaneous Pegzilarginase in Subjects <24 months old with Arginase 1 Deficiency

This study is looking at a new medicine called Pegzilarginase for very young children (under 2 years old) with a rare condition called Arginase 1 Deficiency. Doctors want to see how safe and effective this medicine is at bringing down high levels of a substance called arginine in the blood.