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Crigler-Najjar syndrome type 1

ORPHA:79234Clinical subtypeAutosomal recessiveNeonatal

Ассоциированные гены (1)

UGT1A1
UDP glucuronosyltransferase family 1 member A1
Disease-causing germline mutation(s) (loss of function) in
OMIM: 191740

Фенотипы (14)

Очень частый (80–99%)7
HP:0001080Biliary tract abnormality
HP:0001343Kernicterus
HP:0001392Abnormality of the liver
HP:0003265Neonatal hyperbilirubinemia
HP:0006579Prolonged neonatal jaundice
HP:0008282Unconjugated hyperbilirubinemia
HP:0008947Floppy infant
Периодический (5–29%)7
HP:0000365Hearing impairment
HP:0000750Delayed speech and language development
HP:0001249Intellectual disability
HP:0001250Seizure
HP:0001337Tremor
HP:0002354Memory impairment
HP:0012246Oculomotor nerve palsy

Эпидемиология (1)

Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы