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Waardenburg syndrome type 1

ORPHA:894Clinical subtypeAutosomal dominantNeonatal

Ассоциированные гены (1)

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

Фенотипы (32)

Очень частый (80–99%)16
HP:0000303Mandibular prognathia
HP:0000365Hearing impairment
HP:0000478Abnormality of the eye
HP:0000504Abnormality of vision
HP:0000506Telecanthus
HP:0000574Thick eyebrow
HP:0000632Lacrimation abnormality
HP:0001053Hypopigmented skin patches
HP:0001100Heterochromia iridis
HP:0002211White forelock
HP:0002226White eyebrow
HP:0002227White eyelashes
HP:0003196Short nose
HP:0005599Hypopigmentation of hair
HP:0008527Congenital sensorineural hearing impairment
HP:0011364White hair
Частый (30–79%)6
HP:0000430Underdeveloped nasal alae
HP:0000431Wide nasal bridge
HP:0000664Synophrys
HP:0001595Abnormality of the hair
HP:0002216Premature graying of hair
HP:0010804Tented upper lip vermilion
Периодический (5–29%)10
HP:0000175Cleft palate
HP:0000204Cleft upper lip
HP:0000486Strabismus
HP:0000508Ptosis
HP:0000912Sprengel anomaly
HP:0002251Aganglionic megacolon
HP:0002414Spina bifida
HP:0002435Meningocele
HP:0002650Scoliosis
HP:0030680Abnormal cardiovascular system morphology

Эпидемиология (2)

Point prevalence
1-9 / 100 000
Europe
Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы