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Lymphangioleiomyomatosis

ORPHA:538DiseaseNot applicableAdult

Ассоциированные гены (2)

TSC1
TSC complex subunit 1
Disease-causing somatic mutation(s) in
OMIM: 605284
TSC2
TSC complex subunit 2
Disease-causing somatic mutation(s) in
OMIM: 191092

Фенотипы (36)

Очень частый (80–99%)6
HP:0002091Restrictive ventilatory defect
HP:0002094Dyspnea
HP:0002113Pulmonary infiltrates
HP:0012735Cough
HP:0100749Chest pain
HP:0100763Abnormality of the lymphatic system
Частый (30–79%)11
HP:0000008Abnormal morphology of female internal genitalia
HP:0000790Hematuria
HP:0002027Abdominal pain
HP:0002097Emphysema
HP:0002107Pneumothorax
HP:0002716Lymphadenopathy
HP:0006772Renal angiomyolipoma
HP:0010310Chylothorax
HP:0012798Pulmonary lymphangiomyomatosis
HP:0100750Atelectasis
HP:0100804Ungual fibroma
Периодический (5–29%)19
HP:0000238Hydrocephalus
HP:0000648Optic atrophy
HP:0001000Abnormality of skin pigmentation
HP:0001004Lymphedema
HP:0001250Seizure
HP:0001541Ascites
HP:0001945Fever
HP:0002105Hemoptysis
HP:0002205Recurrent respiratory infections
HP:0002239Gastrointestinal hemorrhage
HP:0005562Multiple renal cysts
HP:0009594Retinal hamartoma
HP:0009721Shagreen patch
HP:0009726Renal neoplasm
HP:0011852Chylopericardium
HP:0012086Abnormal urinary color
HP:0012378Fatigue
HP:0012733Macule
HP:0100543Cognitive impairment

Эпидемиология (13)

Annual incidence
<1 / 1 000 000
Worldwide
Point prevalence
1-9 / 1 000 000
Worldwide
Annual incidence
<1 / 1 000 000
United States
Point prevalence
1-9 / 1 000 000
United States
Annual incidence
<1 / 1 000 000
France
Point prevalence
1-9 / 1 000 000
Germany
Point prevalence
1-9 / 1 000 000
Canada
Point prevalence
1-9 / 1 000 000
United Kingdom
Point prevalence
1-9 / 1 000 000
Switzerland
Point prevalence
1-9 / 1 000 000
New Zealand
Point prevalence
1-9 / 1 000 000
Australia
Point prevalence
1-9 / 1 000 000
Europe
Point prevalence
1-9 / 1 000 000
Netherlands

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы