CTNNB1 NEURODEVELOPMENTAL SYNDROME
Clinical trials for CTNNB1 NEURODEVELOPMENTAL SYNDROME explained in plain language.
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First gene therapy trial launches for rare childhood brain disorder
Disease control Recruiting nowThis study tests a one-time gene therapy given directly into the fluid around the brain in 12 children aged 2-12 with CTNNB1 syndrome, a rare genetic condition causing developmental delays. The goal is to see if the treatment is safe and can improve motor skills, thinking, behavi…
Matched conditions: CTNNB1 NEURODEVELOPMENTAL SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: CTNNB1 Foundation • Aim: Disease control
Last updated May 14, 2026 12:06 UTC
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Dragonfly study aims to unlock mysteries of rare genetic condition
Knowledge-focused Recruiting nowThis study follows 250 children and adults with CTNNB1 neurodevelopmental syndrome to track their mental, physical, and social development over time. By collecting data through assessments and questionnaires, researchers hope to better understand the condition and improve care. T…
Matched conditions: CTNNB1 NEURODEVELOPMENTAL SYNDROME
Sponsor: University Medical Centre Ljubljana • Aim: Knowledge-focused
Last updated May 17, 2026 00:48 UTC