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Mandibulofacial dysostosis-microcephaly syndrome

ORPHA:79113Malformation syndromeAutosomal dominantNeonatal

Ассоциированные гены (1)

EFTUD2
elongation factor Tu GTP binding domain containing 2
Disease-causing germline mutation(s) in
OMIM: 603892

Фенотипы (30)

Очень частый (80–99%)20
HP:0000175Cleft palate
HP:0000243Trigonocephaly
HP:0000272Malar flattening
HP:0000327Hypoplasia of the maxilla
HP:0000347Micrognathia
HP:0000356Abnormality of the outer ear
HP:0000369Low-set ears
HP:0000384Preauricular skin tag
HP:0000582Upslanted palpebral fissure
HP:0000750Delayed speech and language development
HP:0001249Intellectual disability
HP:0003196Short nose
HP:0004322Short stature
HP:0005484Secondary microcephaly
HP:0008551Microtia
HP:0008609Morphological abnormality of the middle ear
HP:0009738Abnormality of the antihelix
HP:0011268Absent tragus
HP:0011272Underdeveloped tragus
HP:0011968Feeding difficulties
Частый (30–79%)7
HP:0000191Accessory oral frenulum
HP:0000286Epicanthus
HP:0000396Overfolded helix
HP:0000413Atresia of the external auditory canal
HP:0000506Telecanthus
HP:0001177Preaxial hand polydactyly
HP:0009748Large earlobe
Периодический (5–29%)3
HP:0000405Conductive hearing impairment
HP:0001250Seizure
HP:0001631Atrial septal defect

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