Ornithine Transcarbamylase Deficiency research hub

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.