CTNNB1 NEURODEVELOPMENTAL SYNDROME
Clinical trials for CTNNB1 NEURODEVELOPMENTAL SYNDROME explained in plain language.
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Brain-Delivered gene therapy offers hope for devastating childhood disorder
Disease control Recruiting nowThis is the first human trial of a gene therapy for children with CTNNB1 syndrome, a severe genetic disorder that affects brain development. Researchers will test if a single dose of a therapy called Urbagen, delivered directly to the brain, is safe and can improve motor function…
Matched conditions: CTNNB1 NEURODEVELOPMENTAL SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: CTNNB1 Foundation • Aim: Disease control
Last updated Mar 19, 2026 14:56 UTC
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Global quest to map rare brain Disorder's journey
Knowledge-focused Recruiting nowThis study aims to understand how CTNNB1 syndrome, a rare genetic condition affecting brain development, changes over a person's lifetime. Researchers will follow 250 children and adults with the condition for five years, tracking their physical, mental, and social development. T…
Matched conditions: CTNNB1 NEURODEVELOPMENTAL SYNDROME
Sponsor: University Medical Centre Ljubljana • Aim: Knowledge-focused
Last updated Mar 25, 2026 14:08 UTC