Weekly shot tested in babies with rare metabolic disorder
Disease control
Completed
This study tested a weekly injection called pegzilarginase in babies under 2 years old with a rare genetic disorder called arginase 1 deficiency. The goal was to see if the treatment safely lowers high levels of a harmful substance called arginine in the blood. Three very young c…
Phase: PHASE3 • Sponsor: Immedica Pharma AB • Aim: Disease control
Last updated Apr 02, 2026 08:42 UTC