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Campomelic dysplasia

ORPHA:140Malformation syndromeAutosomal dominantAntenatal, Neonatal

Ассоциированные гены (1)

SOX9
SRY-box transcription factor 9
Disease-causing germline mutation(s) (loss of function) in
OMIM: 608160

Фенотипы (38)

Очень частый (80–99%)21
HP:0000175Cleft palate
HP:0000256Macrocephaly
HP:0000347Micrognathia
HP:0000470Short neck
HP:0000774Narrow chest
HP:000087811 pairs of ribs
HP:0001601Laryngomalacia
HP:0002093Respiratory insufficiency
HP:0002650Scoliosis
HP:0002757Recurrent fractures
HP:0002779Tracheomalacia
HP:0002786Tracheobronchomalacia
HP:0002827Hip dislocation
HP:0002982Tibial bowing
HP:0003026Short long bone
HP:0003038Fibular hypoplasia
HP:0006487Bowing of the long bones
HP:0006584Small abnormally formed scapulae
HP:0008477Poorly ossified cervical vertebrae
HP:0008821Hypoplastic inferior ilia
HP:0012368Flat face
Частый (30–79%)9
HP:0000037Male pseudohermaphroditism
HP:0000062Ambiguous genitalia
HP:0000316Hypertelorism
HP:0000369Low-set ears
HP:0000520Proptosis
HP:0001762Talipes equinovarus
HP:0002980Femoral bowing
HP:0004322Short stature
HP:0010781Skin dimple
Периодический (5–29%)8
HP:0000126Hydronephrosis
HP:0000365Hearing impairment
HP:0002119Ventriculomegaly
HP:0002808Kyphosis
HP:0004408Abnormality of the sense of smell
HP:0005280Depressed nasal bridge
HP:0007036Hypoplasia of olfactory tract
HP:0030680Abnormal cardiovascular system morphology

Эпидемиология (2)

Point prevalence
<1 / 1 000 000
Worldwide
Prevalence at birth
1-9 / 100 000
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы