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WAGR syndrome

ORPHA:893Malformation syndromeAutosomal dominantAntenatal, Neonatal

Ассоциированные гены (3)

WT1
WT1 transcription factor
Role in the phenotype of
OMIM: 607102
PAX6
paired box 6
Role in the phenotype of
OMIM: 607108
BDNF
brain derived neurotrophic factor
Role in the phenotype of
OMIM: 113505

Фенотипы (18)

Очень частый (80–99%)1
HP:0008053Aplasia/Hypoplasia of the iris
Частый (30–79%)12
HP:0000028Cryptorchidism
HP:0000232Everted lower lip vermilion
HP:0000252Microcephaly
HP:0000347Micrognathia
HP:0000364Hearing abnormality
HP:0000505Visual impairment
HP:0000508Ptosis
HP:0000518Cataract
HP:0000639Nystagmus
HP:0001249Intellectual disability
HP:0004322Short stature
HP:0100627Displacement of the urethral meatus
Периодический (5–29%)5
HP:0000062Ambiguous genitalia
HP:0000501Glaucoma
HP:0001513Obesity
HP:0002650Scoliosis
HP:0007299Dysfunction of lateral corticospinal tracts

Эпидемиология (2)

Prevalence at birth
1-9 / 1 000 000
Worldwide
Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы