This LTFU is being conducted to assess long-term safety and durability of response in participants dosed with IP in a parent protocol, and to collect longitudinal natural history in enrolled but not dosed participants who also participated in a parent protocol.
- Ornithine Carbamoyltransferase Deficiency Disease
- Urea Cycle Disorders, Inborn
- Amino Acid Metabolism, Inborn Errors
- Brain Diseases
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Central Nervous System Diseases
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Hyperammonemia
- Liver Diseases
- Metabolic Diseases
- Metabolism, Inborn Errors
- Nervous System Diseases
Age: 7 - 15 months
Gender: All