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Childhood-onset motor and cognitive regression syndrome with extrapyramidal movement disorder

MONDO:0044701

Also known as: UBTF-related disorder, CONDBA, neurodegeneration, childhood-onset, with brain atrophy

16 clinical trials for this condition and its sub-types.

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Broader categories

Disease (618) Nervous system disorder (217) Hereditary disease (172) Movement disorder (89) Human disease (14) Developmental defect during embryogenesis (7) Central nervous system malformation (5) Hereditary neurological disease (5) Disease of genetic or genomic mechanism (2) Congenital nervous system disorder (1)
Trials to join now! 11 Not yet finished but already full! 1 Completed 4
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  • Custom Gene-Targeting drug offers hope for one child with fatal brain disorder

    Disease control Ongoing

    This study tests a custom-made drug for one child with a rare, severe brain disease called CONDBA, caused by a specific gene mutation. The drug aims to slow or stop brain damage by targeting the faulty gene. Researchers will track changes in movement, coordination, and quality of…

    Phase: PHASE1, PHASE2 • Sponsor: n-Lorem Foundation • Aim: Disease control

    Last updated Jun 27, 2026 12:32 UTC

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