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Severe feeding difficulties-failure to thrive-microcephaly due to ASXL3 deficiency syndrome

MONDO:0014205

Also known as: ASXL3-Related Disorder, Bainbridge-Roppers syndrome, severe feeding difficulties-failure to thrive-microcephaly due to ASXL3 deficiency syndrome, BAINBRIDGE-ROPERS syndrome, BRPS

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

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Broader categories

Disease (618) Hereditary disease (172) Human disease (14) Developmental defect during embryogenesis (7) Disease of genetic or genomic mechanism (2) Multiple congenital anomalies/dysmorphic syndrome (1) Multiple congenital anomalies/dysmorphic syndrome-intellectual disability (1) Disease by developmental or physiological process (0) Disease by etiologic mechanism (0) Disorder of development or morphogenesis (0)
Trials to join now! 3 Not yet finished but already full! 1 Completed 2
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  • One patient, one custom drug: a bold experiment for a rare syndrome

    Disease control Ongoing

    This study tests a custom-made drug called an antisense oligonucleotide (ASO) designed specifically for one person with Bainbridge-Ropers syndrome, a rare genetic condition that causes developmental delays. The drug aims to correct the effects of a specific ASXL3 gene mutation. T…

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

    Last updated Jun 27, 2026 07:52 UTC

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