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 study tests a new gene therapy for children aged 2-12 with CTNNB1 neurodevelopmental syndrome, a rare genetic disorder causing severe developmental delays. Twelve participants will receive a single dose of the therapy directly into the brain fluid, along with lifelong immune…
Matched conditions: CTNNB1 NEURODEVELOPMENTAL SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: CTNNB1 Foundation • Aim: Disease control
Last updated May 26, 2026 08:51 UTC
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Dragonfly study launches to map rare genetic Syndrome's journey
Knowledge-focused Recruiting nowThis study follows 250 children and adults with CTNNB1 neurodevelopmental syndrome to track their mental, physical, and social development over time. Researchers will collect data through assessments and questionnaires to better understand the condition and improve care. The stud…
Matched conditions: CTNNB1 NEURODEVELOPMENTAL SYNDROME
Sponsor: University Medical Centre Ljubljana • Aim: Knowledge-focused
Last updated May 26, 2026 08:30 UTC