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Frontonasal dysplasia-severe microphthalmia-severe facial clefting syndrome

ORPHA:306542Malformation syndromeAutosomal recessiveAntenatal, Neonatal

Ассоциированные гены (1)

ALX1
ALX homeobox 1
Disease-causing germline mutation(s) (loss of function) in
OMIM: 601527

Фенотипы (29)

Частый (30–79%)25
HP:0000175Cleft palate
HP:0000286Epicanthus
HP:0000316Hypertelorism
HP:0000327Hypoplasia of the maxilla
HP:0000349Widow's peak
HP:0000358Posteriorly rotated ears
HP:0000384Preauricular skin tag
HP:0000405Conductive hearing impairment
HP:0000430Underdeveloped nasal alae
HP:0000431Wide nasal bridge
HP:0000508Ptosis
HP:0000518Cataract
HP:0000568Microphthalmia
HP:0000625Eyelid coloboma
HP:0000653Sparse eyelashes
HP:0001156Brachydactyly
HP:0001274Agenesis of corpus callosum
HP:0002006Facial cleft
HP:0005258Pectoral muscle hypoplasia/aplasia
HP:0005466Hypoplasia of the frontal bone
HP:0009119Aplasia/Hypoplasia of the frontal sinuses
HP:0011803Bifid nose
HP:0040019Finger clinodactyly
HP:0045075Sparse eyebrow
HP:0100490Camptodactyly of finger
Периодический (5–29%)4
HP:0001249Intellectual disability
HP:0001636Tetralogy of Fallot
HP:0004423Cranium bifidum occultum
HP:0006931Lipoma of corpus callosum

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