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Osteosclerotic bone dysplasia

ORPHA:1832Malformation syndromeAutosomal recessiveAntenatal, Neonatal

Ассоциированные гены (1)

FAM20C
FAM20C golgi associated secretory pathway kinase
Disease-causing germline mutation(s) (loss of function) in
OMIM: 611061

Фенотипы (20)

Очень частый (80–99%)11
HP:0000239Large fontanelles
HP:0000252Microcephaly
HP:0000270Delayed cranial suture closure
HP:0000278Retrognathia
HP:0000347Micrognathia
HP:0000358Posteriorly rotated ears
HP:0000369Low-set ears
HP:0000457Depressed nasal ridge
HP:0000463Anteverted nares
HP:0000470Short neck
HP:0009099Median cleft palate
Частый (30–79%)9
HP:0000169Gingival fibromatosis
HP:0000212Gingival overgrowth
HP:0000520Proptosis
HP:0001511Intrauterine growth retardation
HP:0002094Dyspnea
HP:0002098Respiratory distress
HP:0002878Respiratory failure
HP:0003196Short nose
HP:0009939Mandibular aplasia

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