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Adenine phosphoribosyltransferase deficiency

ORPHA:976DiseaseAutosomal recessiveAdolescent, Adult, Childhood, Elderly, Infancy

Ассоциированные гены (1)

APRT
adenine phosphoribosyltransferase
Disease-causing germline mutation(s) (loss of function) in
OMIM: 102600

Фенотипы (18)

Очень частый (80–99%)1
HP:0012379Abnormal enzyme/coenzyme activity
Частый (30–79%)7
HP:0000083Renal insufficiency
HP:0000093Proteinuria
HP:0000787Nephrolithiasis
HP:0000822Hypertension
HP:0001919Acute kidney injury
HP:0012622Chronic kidney disease
HP:0100518Dysuria
Периодический (5–29%)10
HP:0000010Recurrent urinary tract infections
HP:0000016Urinary retention
HP:0000019Urinary hesitancy
HP:0000791Uric acid nephrolithiasis
HP:0003774Stage 5 chronic kidney disease
HP:0005110Atrial fibrillation
HP:0011848Abdominal colic
HP:0012587Macroscopic hematuria
HP:0030157Flank pain
HP:0100520Oliguria

Эпидемиология (4)

Point prevalence
1-9 / 100 000
Specific population
Point prevalence
1-9 / 100 000
Japan
Point prevalence
1-9 / 100 000
Iceland
Point prevalence
1-9 / 100 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы