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Generalized pseudohypoaldosteronism type 1

ORPHA:171876Clinical subtypeAutosomal recessiveInfancy, Neonatal

Ассоциированные гены (3)

SCNN1A
sodium channel epithelial 1 subunit alpha
Disease-causing germline mutation(s) (loss of function) in
OMIM: 600228
SCNN1B
sodium channel epithelial 1 subunit beta
Disease-causing germline mutation(s) (loss of function) in
OMIM: 600760
SCNN1G
sodium channel epithelial 1 subunit gamma
Disease-causing germline mutation(s) (loss of function) in
OMIM: 600761

Фенотипы (22)

Очень частый (80–99%)6
HP:0000848Increased circulating renin level
HP:0001942Metabolic acidosis
HP:0002153Hyperkalemia
HP:0002902Hyponatremia
HP:0011740Glucocortocoid-insensitive primary hyperaldosteronism
HP:0040085Abnormal circulating aldosterone
Частый (30–79%)5
HP:0001531Failure to thrive in infancy
HP:0001944Dehydration
HP:0002013Vomiting
HP:0031274Hypovolemic shock
HP:0200117Recurrent upper and lower respiratory tract infections
Периодический (5–29%)11
HP:0001047Atopic dermatitis
HP:0001081Cholelithiasis
HP:0001824Weight loss
HP:0002754Osteomyelitis
HP:0003508Proportionate short stature
HP:0008872Feeding difficulties in infancy
HP:0011110Recurrent tonsillitis
HP:0011675Arrhythmia
HP:0012735Cough
HP:0030828Wheezing
HP:0200039Pustule

Эпидемиология (1)

Prevalence at birth
1-9 / 1 000 000
United Kingdom

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы