Arginase I deficiency

Clinical trials for Arginase I deficiency explained in plain language.

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Completed 1

Can an enzyme replacement tame a rare metabolic disorder?

Disease control Completed

This phase 3 trial is testing whether pegzilarginase, an enzyme replacement therapy, can lower plasma arginine levels and improve mobility in children and adults with arginase 1 deficiency, a rare genetic condition that causes high arginine levels and neurological problems. Parti…

Matched conditions: ARGINASE I DEFICIENCY

Phase 3 • Sponsor: Immedica Pharma AB • Aim: Disease control

Last updated Aug 23, 2026 00:00 UTC

Arginase I deficiency

Get alerted when new Arginase I deficiency trials appear

Can an enzyme replacement tame a rare metabolic disorder?