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Anophthalmia/microphthalmia-esophageal atresia syndrome

ORPHA:77298Malformation syndromeAutosomal dominant, Not applicableAntenatal, Neonatal

Ассоциированные гены (1)

SOX2
SRY-box transcription factor 2
Disease-causing germline mutation(s) in
OMIM: 184429

Фенотипы (22)

Очень частый (80–99%)4
HP:0000528Anophthalmia
HP:0000568Microphthalmia
HP:0002032Esophageal atresia
HP:0002575Tracheoesophageal fistula
Частый (30–79%)4
HP:0000028Cryptorchidism
HP:0000365Hearing impairment
HP:0000572Visual loss
HP:0001274Agenesis of corpus callosum
Периодический (5–29%)14
HP:0000047Hypospadias
HP:0000238Hydrocephalus
HP:0000612Iris coloboma
HP:0000647Sclerocornea
HP:000087811 pairs of ribs
HP:0001249Intellectual disability
HP:0001263Global developmental delay
HP:0001360Holoprosencephaly
HP:0001510Growth delay
HP:0001629Ventricular septal defect
HP:0001643Patent ductus arteriosus
HP:0002937Hemivertebrae
HP:0003468Abnormal vertebral morphology
HP:0008736Hypoplasia of penis

Эпидемиология (2)

Cases/families
Worldwide
Point prevalence
<1 / 1 000 000
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы