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Agnathia-holoprosencephaly-situs inversus syndrome

ORPHA:990Malformation syndromeAutosomal dominant, Autosomal recessive, Not applicableAntenatal, Neonatal

Ассоциированные гены (2)

OTX2
orthodenticle homeobox 2
Modifying germline mutation in
OMIM: 600037
PRRX1
paired related homeobox 1
Disease-causing germline mutation(s) (loss of function) in
OMIM: 167420

Фенотипы (19)

Очень частый (80–99%)19
HP:0000358Posteriorly rotated ears
HP:0000160Narrow mouth
HP:0000171Microglossia
HP:0000478Abnormality of the eye
HP:0001274Agenesis of corpus callosum
HP:0001291Abnormal cranial nerve morphology
HP:0001360Holoprosencephaly
HP:0001561Polyhydramnios
HP:0001696Situs inversus totalis
HP:0002098Respiratory distress
HP:0007360Aplasia/Hypoplasia of the cerebellum
HP:0008736Hypoplasia of penis
HP:0009914Cyclopia
HP:0009924Aplasia/Hypoplasia involving the nose
HP:0009939Mandibular aplasia
HP:0011386Narrow internal auditory canal
HP:0100596Absent nares
HP:0100663Synotia
HP:0100840Aplasia/Hypoplasia of the eyebrow

Эпидемиология (1)

Point prevalence
<1 / 1 000 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы