Fundacija CTNNB1 Ustanova Za Raziskave Na Podrocju Genske Terapije

Research activity is centered on CTNNB1 syndrome, with attention to the pediatric population affected by this rare neurodevelopmental disorder. The sponsor’s clinical focus includes gene therapy approaches designed for central nervous system delivery.

• CTNNB1-related neurological disease
• pediatric neurogenetic conditions
• rare disease therapeutics

The sponsor’s trial portfolio is directed toward conditions involving impaired brain development and neurological function.