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Fibrochondrogenesis

ORPHA:2021DiseaseAutosomal dominant, Autosomal recessiveNeonatal

Ассоциированные гены (2)

COL11A1
collagen type XI alpha 1 chain
Disease-causing germline mutation(s) (loss of function) in
OMIM: 120280
COL11A2
collagen type XI alpha 2 chain
Disease-causing germline mutation(s) in
OMIM: 120290

Фенотипы (29)

Очень частый (80–99%)14
HP:0000260Wide anterior fontanel
HP:0000311Round face
HP:0000470Short neck
HP:0000520Proptosis
HP:0000772Abnormal rib morphology
HP:0000773Short ribs
HP:0000774Narrow chest
HP:0000885Broad ribs
HP:0000940Abnormal diaphysis morphology
HP:0000944Abnormal metaphysis morphology
HP:0001156Brachydactyly
HP:0001591Bell-shaped thorax
HP:0003312Abnormal form of the vertebral bodies
HP:0004322Short stature
Частый (30–79%)10
HP:0000160Narrow mouth
HP:0000175Cleft palate
HP:0000364Hearing abnormality
HP:0000369Low-set ears
HP:0000463Anteverted nares
HP:0000494Downslanted palpebral fissures
HP:0000882Hypoplastic scapulae
HP:0001804Hypoplastic fingernail
HP:0002093Respiratory insufficiency
HP:0005280Depressed nasal bridge
Периодический (5–29%)5
HP:0000316Hypertelorism
HP:0001357Plagiocephaly
HP:0001539Omphalocele
HP:0002983Micromelia
HP:0100490Camptodactyly of finger

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