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Reticular dysgenesis

ORPHA:33355DiseaseAutosomal recessiveInfancy, Neonatal

Ассоциированные гены (1)

AK2
adenylate kinase 2
Disease-causing germline mutation(s) (loss of function) in
OMIM: 103020

Фенотипы (20)

Очень частый (80–99%)13
HP:0000365Hearing impairment
HP:0000389Chronic otitis media
HP:0001874Abnormality of neutrophils
HP:0001882Leukopenia
HP:0001903Anemia
HP:0002014Diarrhea
HP:0002205Recurrent respiratory infections
HP:0003287Abnormality of mitochondrial metabolism
HP:0004313Decreased circulating antibody level
HP:0004430Severe combined immunodeficiency
HP:0005374Cellular immunodeficiency
HP:0010515Aplasia/Hypoplasia of the thymus
HP:0100806Sepsis
Частый (30–79%)4
HP:0001508Failure to thrive
HP:0001824Weight loss
HP:0001945Fever
HP:0002024Malabsorption
Периодический (5–29%)3
HP:0000988Skin rash
HP:0001944Dehydration
HP:0200042Skin ulcer

Эпидемиология (2)

Annual incidence
<1 / 1 000 000
Europe
Point prevalence
<1 / 1 000 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы