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Primary hyperoxaluria type 1

ORPHA:93598Clinical subtypeAutosomal recessiveAll ages

Ассоциированные гены (1)

AGXT
alanine--glyoxylate aminotransferase
Disease-causing germline mutation(s) in
OMIM: 604285

Фенотипы (19)

Очень частый (80–99%)7
HP:0000121Nephrocalcinosis
HP:0000787Nephrolithiasis
HP:0001903Anemia
HP:0001942Metabolic acidosis
HP:0003159Hyperoxaluria
HP:0003761Calcinosis
HP:0011021Abnormality of circulating enzyme level
Частый (30–79%)4
HP:0100518Dysuria
HP:0000790Hematuria
HP:0001508Failure to thrive
HP:0012213Decreased glomerular filtration rate
Периодический (5–29%)4
HP:0000010Recurrent urinary tract infections
HP:0000805Enuresis
HP:0000924Abnormality of the skeletal system
HP:0003774Stage 5 chronic kidney disease
Очень редкий (1–4%)4
HP:0000164Abnormality of the dentition
HP:0001297Stroke
HP:0001939Abnormality of metabolism/homeostasis
HP:0002621Atherosclerosis

Эпидемиология (6)

Annual incidence
<1 / 1 000 000
Netherlands
Point prevalence
1-9 / 1 000 000
Netherlands
Prevalence at birth
1-9 / 100 000
Switzerland
Point prevalence
1-9 / 1 000 000
Switzerland
Annual incidence
1-9 / 1 000 000
France
Point prevalence
1-9 / 1 000 000
France

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы