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MYH9-related syndromic thrombocytopenia

ORPHA:182050DiseaseAutosomal dominantAdolescent, Adult, Childhood, Infancy, Neonatal

Ассоциированные гены (1)

MYH9
myosin heavy chain 9
Disease-causing germline mutation(s) in
OMIM: 160775

Фенотипы (16)

Очень частый (80–99%)1
HP:0001905Congenital thrombocytopenia
Частый (30–79%)14
HP:0000083Renal insufficiency
HP:0000093Proteinuria
HP:0000112Nephropathy
HP:0000123Nephritis
HP:0000132Menorrhagia
HP:0000407Sensorineural hearing impairment
HP:0000978Bruising susceptibility
HP:0001902Giant platelets
HP:0002910Elevated circulating hepatic transaminase concentration
HP:0003010Prolonged bleeding time
HP:0004406Spontaneous, recurrent epistaxis
HP:0007819Presenile cataracts
HP:0008264Neutrophil inclusion bodies
HP:0011877Increased mean platelet volume
Очень редкий (1–4%)1
HP:0001658Myocardial infarction

Эпидемиология (2)

Point prevalence
1-9 / 1 000 000
Italy
Point prevalence
1-9 / 1 000 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы