Canavan disease, Canavan Disease research hub

Phase 1/2, Open Label, Sequential Cohort Study of a Single Intracranial Dose of rAAVOlig001-ASPA Gene Therapy for Treatment of Children with Typical Canavan Disease

This study looks at a new gene therapy for children with Canavan disease. Researchers want to see if a single treatment helps reduce a harmful chemical in the brain and improves movement and development. It's an early-stage study, meaning it's one of the first times this treatment is being tried in people.