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Craniometaphyseal dysplasia

ORPHA:1522Malformation syndromeAutosomal dominant, Autosomal recessiveChildhood

Ассоциированные гены (2)

ANKH
ANKH inorganic pyrophosphate transport regulator
Disease-causing germline mutation(s) (gain of function) in
OMIM: 605145
GJA1
gap junction protein alpha 1
Disease-causing germline mutation(s) in
OMIM: 121014

Фенотипы (13)

Очень частый (80–99%)6
HP:0000316Hypertelorism
HP:0000431Wide nasal bridge
HP:0000944Abnormal metaphysis morphology
HP:0004493Craniofacial hyperostosis
HP:0005280Depressed nasal bridge
HP:0011002Osteopetrosis
Частый (30–79%)2
HP:0000506Telecanthus
HP:0002652Skeletal dysplasia
Периодический (5–29%)5
HP:0000405Conductive hearing impairment
HP:0000407Sensorineural hearing impairment
HP:0000505Visual impairment
HP:0001291Abnormal cranial nerve morphology
HP:0010628Facial palsy

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