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Cerebrofaciothoracic dysplasia

ORPHA:1394Malformation syndromeAutosomal recessiveAntenatal, Neonatal

Ассоциированные гены (1)

TMCO1
transmembrane and coiled-coil domains 1
Disease-causing germline mutation(s) (loss of function) in
OMIM: 614123

Фенотипы (35)

Очень частый (80–99%)15
HP:0000154Wide mouth
HP:0000248Brachycephaly
HP:0000289Broad philtrum
HP:0000316Hypertelorism
HP:0000445Wide nose
HP:0000470Short neck
HP:0000574Thick eyebrow
HP:0000774Narrow chest
HP:0000892Bifid ribs
HP:0000902Rib fusion
HP:0001249Intellectual disability
HP:0002079Hypoplasia of the corpus callosum
HP:0002937Hemivertebrae
HP:0011800Midface retrusion
HP:0000358Posteriorly rotated ears
Частый (30–79%)18
HP:0000256Macrocephaly
HP:0000286Epicanthus
HP:0000486Strabismus
HP:0000494Downslanted palpebral fissures
HP:0000664Synophrys
HP:0000912Sprengel anomaly
HP:0001320Cerebellar vermis hypoplasia
HP:0001561Polyhydramnios
HP:0002119Ventriculomegaly
HP:0002120Cerebral cortical atrophy
HP:0002162Low posterior hairline
HP:0002208Coarse hair
HP:0002650Scoliosis
HP:0003196Short nose
HP:0003422Vertebral segmentation defect
HP:0004322Short stature
HP:0010720Abnormal hair pattern
HP:0100790Hernia
Периодический (5–29%)2
HP:0000175Cleft palate
HP:0000204Cleft upper lip

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