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Ornithine Transcarbamylase Deficiency research hub
2 indexed studies · 2 currently recruiting
- recruitingPHASE1, PHASE2
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
This study is testing a new gene therapy called ECUR-506 for baby boys under 9 months old with a severe genetic condition called OTC deficiency. It's an early-stage study looking at how safe the treatment is and if it helps.
United States · Australia · Spain - recruiting
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
This study is checking on the long-term well-being and health progress of children who previously took part in a study for a rare liver condition called Ornithine Transcarbamylase Deficiency (OTCD). It also looks at how the condition naturally develops in those who didn't receive the study treatment.
United Kingdom