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Craniofacial-deafness-hand syndrome

ORPHA:1529Malformation syndromeAutosomal dominantNeonatal

Ассоциированные гены (1)

PAX3
paired box 3
Disease-causing germline mutation(s) in
OMIM: 606597

Фенотипы (17)

Очень частый (80–99%)16
HP:0000160Narrow mouth
HP:0000275Narrow face
HP:0000316Hypertelorism
HP:0000327Hypoplasia of the maxilla
HP:0000407Sensorineural hearing impairment
HP:0000457Depressed nasal ridge
HP:0000494Downslanted palpebral fissures
HP:0000564Lacrimal duct atresia
HP:0000581Blepharophimosis
HP:0003019Abnormality of the wrist
HP:0003049Ulnar deviation of the wrist
HP:0003196Short nose
HP:0005280Depressed nasal bridge
HP:0009465Ulnar deviation of finger
HP:0009924Aplasia/Hypoplasia involving the nose
HP:0012368Flat face
Частый (30–79%)1
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