Records containing: Ornithine Transcarbamylase Deficiency
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CLINICAL_TRIALS study record update · 2026-04-27Noninvasive Biomarkers of Hepatic Fibrosis in Urea Cycle Disorders
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CLINICAL_TRIALS study record update · 2026-04-15A 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
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CLINICAL_TRIALS study record update · 2025-09-26Phase 2, Randomized, Double-Blind, Placebo-Controlled, Nested Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of ARCT-810 in Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency
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CLINICAL_TRIALS study record update · 2025-08-12Universal Genomic Newborn Screening in the Wallonia-Brussels Federation: Baby Detect
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CLINICAL_TRIALS study record update · 2025-07-20Hepatic Histopathology in Urea Cycle Disorders
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CLINICAL_TRIALS study record update · 2025-04-17Noninvasive Biomarkers of Hepatic Fibrosis in Urea Cycle Disorders
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CLINICAL_TRIALS study record update · 2025-04-04Early Check: A Collaborative Innovation to Facilitate Pre-Symptomatic Clinical Trials in Newborns
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CLINICAL_TRIALS study record update · 2025-03-17A Phase 2a, Open-label, Multiple Ascending Dose Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency
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CLINICAL_TRIALS study record update · 2025-02-03Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
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CLINICAL_TRIALS study record update · 2024-08-02A Phase 1b Randomized, Double Blinded, Placebo Controlled, Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Single Doses of ARCT-810 in Clinically Stable Patients With Ornithine Transcarbamylase Deficiency
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CLINICAL_TRIALS study record update · 2023-11-08A Phase 1 Randomized, Double Blinded, Placebo Controlled, Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Single Doses of ARCT-810 in Healthy Adult Subjects
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CLINICAL_TRIALS study record update · 2023-11-07Phase I/II Open Label, Multicentre Clinical Trial to Assess Safety and Efficacy of AAVLK03hOTC for Paediatric Patients With Ornithine Transcarbamylase Deficiency.
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CLINICAL_TRIALS study record update · 2023-06-18Introduction of Tandem Mass Spectrometry (MS/MS) Technology in the Program of Selective Screening of Hereditary Metabolic Diseases in Kazakhstan
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CLINICAL_TRIALS study record update · 2023-06-12Observational Clinical Study of Anti-AAV Seroprevalence in Subjects With Ornithine Transcarbamylase Deficiency, Glycogen Storage Disorder Type Ia, and Wilson Disease
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CLINICAL_TRIALS study record update · 2022-02-18A Study to Characterize Rate of Ureagenesis Utilizing Oral [1-13C] Sodium Acetate in the Spectrum of Severity of Patients With Ornithine Transcarbamylase (OTC) Deficiency
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CLINICAL_TRIALS study record update · 2020-03-30A Single-Center Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve From Time Zero to 24 Hours and Rate of Ureagenesis in Healthy Adult Subjects
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CLINICAL_TRIALS study record update · 2019-09-17A Phase 1/2 Single Ascending Dose Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenously Administered MRT5201 in Subjects With Ornithine Transcarbamylase Deficiency
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CLINICAL_TRIALS study record update · 2017-04-14Investigation of Brain Nitrogen in Partial Ornithine Transcarbamylase Deficiency (OTCD) Using 1 H MRS, DTI, and fMRI
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CLINICAL_TRIALS study record update · 2016-02-08Open, Prospective, Uncontrolled, Multicentre Study to Evaluate The Safety and Efficacy of Multiple Applications of Liver Cell Suspension Into The Portal Vein in Children With Urea Cycle Disorders (UCDs)
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CLINICAL_TRIALS study record update · 2015-05-29Assessing Neural Mechanisms of Injury in Inborn Errors of Urea Metabolism Using Structural MRI, Functional MRI, and Magnetic Resonance Spectroscopy