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Waardenburg syndrome type 3

ORPHA:896Clinical subtypeAutosomal dominant, Autosomal recessiveNeonatal

Ассоциированные гены (1)

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

Фенотипы (22)

Очень частый (80–99%)13
HP:0000252Microcephaly
HP:0000271Abnormality of the face
HP:0000365Hearing impairment
HP:0000446Narrow nasal bridge
HP:0000494Downslanted palpebral fissures
HP:0000574Thick eyebrow
HP:0000581Blepharophimosis
HP:0001167Abnormality of finger
HP:0001387Joint stiffness
HP:0002817Abnormality of the upper limb
HP:0005048Synostosis of carpal bones
HP:0010554Cutaneous finger syndactyly
HP:0010804Tented upper lip vermilion
Частый (30–79%)3
HP:0000506Telecanthus
HP:0011364White hair
HP:0100750Atelectasis
Периодический (5–29%)6
HP:0001063Acrocyanosis
HP:0001249Intellectual disability
HP:0001258Spastic paraplegia
HP:0001631Atrial septal defect
HP:0002779Tracheomalacia
HP:0100490Camptodactyly of finger

Эпидемиология (1)

Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы