CTNNB1 NEURODEVELOPMENTAL SYNDROME
Clinical trials for CTNNB1 NEURODEVELOPMENTAL SYNDROME explained in plain language.
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First gene therapy for rare brain disorder begins testing in kids
Disease control Recruiting nowThis early-stage trial tests a gene therapy called Urbagen in 12 children aged 2-12 with CTNNB1 neurodevelopmental syndrome, a rare genetic condition causing motor and cognitive delays. The therapy is given as a single infusion into the brain fluid, along with immunosuppressant d…
Matched conditions: CTNNB1 NEURODEVELOPMENTAL SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: CTNNB1 Foundation • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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Dragonfly study launches to map rare genetic syndrome
Knowledge-focused Recruiting nowThe Dragonfly study is an international observational project tracking the development of 250 children and adults with CTNNB1 neurodevelopmental syndrome. Researchers will collect medical history, perform neurological exams, and use questionnaires to understand how symptoms and a…
Matched conditions: CTNNB1 NEURODEVELOPMENTAL SYNDROME
Sponsor: University Medical Centre Ljubljana • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:51 UTC