Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn

Ornithine Transcarbamylase Deficiency

Study for Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810 Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn Hepatic Histopathology in Urea Cycle Disorders Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency Liver Disease in Urea Cycle Disorders Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With OTC Deficiency Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects The NIH UNI Study: Urea Cycle Disorders, Nutrition and Immunity Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset OTC Deficiency Neurologic Injuries in Adults With Urea Cycle Disorders Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency Investigation of Brain Nitrogen in Partial Ornithine Transcarbamylase Deficiency (OTCD) Using 1 H MRS, DTI, and fMRI Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Deficiency Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency