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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

13 clinical trials for this condition and its sub-types, 1 tagged with Childhood-onset motor and cognitive regression syndrome with extrapyramidal movement disorder itself.

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Where it sits in the disease tree

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Part of

↑ Hereditary neurological disease (5791) ↑ Movement disorder (805) ↑ Congenital nervous system disorder (287) ↑ Multiple congenital anomalies/dysmorphic syndrome-intellectual disability (59) ↑ Central nervous system malformation (51)

Including sub-types (13) Tagged with Childhood-onset motor and cognitive regression syndrome with extrapyramidal movement disorder (1)

Not yet finished but already full! 1

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 1/2 • Sponsor: n-Lorem Foundation • Aim: Disease control

Last updated Jun 27, 2026 12:32 UTC