Ornithine Transcarbamylase Deficiency
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CLINICAL_TRIALS research · 2026-04-22Clinical Trial Update: Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous OTC Genotype [Phase 1]
Phase 1 clinical trial update for CMP-CPS-001 in healthy volunteers and individuals with abnormal heterozygous OTC genotype; the study is ac…
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CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency [Phase 1, Phase 2]
This clinical trial update describes an open-label Phase 1/2 study evaluating ECUR-506 in male infants under 9 months with neonatal-onset Or…
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CLINICAL_TRIALS research · 2026-02-17Clinical Trial Update: Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency [Phase 3]
Clinical trial update indicating that Ultragenyx's DTX301 AAV-mediated gene transfer for ornithine transcarbamylase deficiency is a Phase 3 …
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CLINICAL_TRIALS research · 2025-04-17Clinical Trial Update: Liver Disease in Urea Cycle Disorders
A clinical trial update indicating ongoing recruitment for a study on liver disease in patients with urea cycle disorders, including OTC def…
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CLINICAL_TRIALS research · 2025-03-17Clinical Trial Update: A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD [Phase 2]
A Phase 2 clinical trial update describing the pharmacodynamics and safety evaluation of ARCT-810 in participants with ornithine transcarbam…
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