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Postaxial acrofacial dysostosis

ORPHA:246Malformation syndromeAutosomal recessiveAntenatal, Neonatal

Ассоциированные гены (1)

DHODH
dihydroorotate dehydrogenase (quinone)
Disease-causing germline mutation(s) (loss of function) in
OMIM: 126064

Фенотипы (20)

Очень частый (80–99%)12
HP:0000272Malar flattening
HP:0000347Micrognathia
HP:0000378Cupped ear
HP:0000494Downslanted palpebral fissures
HP:0000625Eyelid coloboma
HP:0002558Supernumerary nipple
HP:0002984Hypoplasia of the radius
HP:0003022Hypoplasia of the ulna
HP:0007477Abnormal dermatoglyphics
HP:0007651Ectropion of lower eyelids
HP:0008551Microtia
HP:0000358Posteriorly rotated ears
Частый (30–79%)7
HP:0100490Camptodactyly of finger
HP:0000175Cleft palate
HP:0000370Abnormality of the middle ear
HP:0000405Conductive hearing impairment
HP:0006101Finger syndactyly
HP:0030680Abnormal cardiovascular system morphology
HP:0100335Non-midline cleft of the upper lip
Периодический (5–29%)1
HP:0000486Strabismus

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