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Axenfeld-Rieger syndrome

ORPHA:782Malformation syndromeAutosomal dominantInfancy, Neonatal

Ассоциированные гены (3)

PITX2
paired like homeodomain 2
Disease-causing germline mutation(s) (loss of function) in
OMIM: 601542
PITX2
paired like homeodomain 2
Disease-causing germline mutation(s) (gain of function) in
OMIM: 601542
FOXC1
forkhead box C1
Disease-causing germline mutation(s) in
OMIM: 601090

Фенотипы (21)

Очень частый (80–99%)3
HP:0000593Abnormal anterior chamber morphology
HP:0000627Posterior embryotoxon
HP:0008053Aplasia/Hypoplasia of the iris
Частый (30–79%)5
HP:0000232Everted lower lip vermilion
HP:0000365Hearing impairment
HP:0000501Glaucoma
HP:0011800Midface retrusion
HP:0030680Abnormal cardiovascular system morphology
Периодический (5–29%)13
HP:0000047Hypospadias
HP:0000316Hypertelorism
HP:0000327Hypoplasia of the maxilla
HP:0000431Wide nasal bridge
HP:0000506Telecanthus
HP:0000668Hypodontia
HP:0000691Microdontia
HP:0000864Abnormality of the hypothalamus-pituitary axis
HP:0001510Growth delay
HP:0001582Redundant skin
HP:0002025Anal stenosis
HP:0005280Depressed nasal bridge
HP:0011220Prominent forehead

Эпидемиология (1)

Point prevalence
1-9 / 1 000 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы