Records containing: Ornithine Carbamoyltransferase Deficiency (Disorder)

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CLINICAL_TRIALS study record update · 2026-04-15
A Phase 1/2/3 First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of a Single Intravenous (IV) Administration of ECUR-506 in Males Less Than 9 Months of Age With Genetically Confirmed Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency