ORNITHINE TRANSCARBAMYLASE DEFICIENCY

Clinical trials for ORNITHINE TRANSCARBAMYLASE DEFICIENCY explained in plain language.

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Trials to join now! 1 Terminated 1

One-Shot gene therapy aims to cure devastating baby disease

⭐️ CURE ⭐️ Recruiting now

This study is testing a single-dose gene therapy called ECUR-506 in baby boys with a severe, life-threatening genetic disorder called OTC deficiency. The goal is to see if delivering a working copy of the faulty gene directly into the body is safe and can help the body properly p…

Matched conditions: ORNITHINE TRANSCARBAMYLASE DEFICIENCY

Phase: PHASE1, PHASE2 • Sponsor: iECURE, Inc. • Aim: ⭐️ CURE ⭐️

Last updated Mar 30, 2026 14:28 UTC

ORNITHINE TRANSCARBAMYLASE DEFICIENCY

Get alerted when new ORNITHINE TRANSCARBAMYLASE DEFICIENCY trials appear

One-Shot gene therapy aims to cure devastating baby disease