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Netherton syndrome

ORPHA:634DiseaseAutosomal recessiveInfancy, Neonatal

Ассоциированные гены (1)

SPINK5
serine peptidase inhibitor Kazal type 5
Disease-causing germline mutation(s) in
OMIM: 605010

Фенотипы (31)

Очень частый (80–99%)14
HP:0000956Acanthosis nigricans
HP:0000964Eczematoid dermatitis
HP:0001025Urticaria
HP:0001595Abnormality of the hair
HP:0002024Malabsorption
HP:0002099Asthma
HP:0002209Sparse scalp hair
HP:0002213Fine hair
HP:0003212Increased circulating IgE level
HP:0007400Irregular hyperpigmentation
HP:0007479Congenital nonbullous ichthyosiform erythroderma
HP:0008064Ichthyosis
HP:0009886Trichorrhexis nodosa
HP:0100326Immunologic hypersensitivity
Частый (30–79%)6
HP:0001249Intellectual disability
HP:0001250Seizure
HP:0001263Global developmental delay
HP:0002097Emphysema
HP:0002205Recurrent respiratory infections
HP:0004313Decreased circulating antibody level
Периодический (5–29%)11
HP:0000086Ectopic kidney
HP:0000126Hydronephrosis
HP:0000653Sparse eyelashes
HP:0000958Dry skin
HP:0000988Skin rash
HP:0001019Erythroderma
HP:0001944Dehydration
HP:0002719Recurrent infections
HP:0003355Aminoaciduria
HP:0004322Short stature
HP:0045075Sparse eyebrow

Эпидемиология (2)

Prevalence at birth
1-9 / 1 000 000
Europe
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