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Autosomal erythropoietic protoporphyria

ORPHA:79278DiseaseAutosomal dominant, Autosomal recessiveInfancy, Neonatal

Ассоциированные гены (1)

FECH
ferrochelatase
Disease-causing germline mutation(s) (loss of function) in
OMIM: 612386

Фенотипы (10)

Очень частый (80–99%)4
HP:0000989Pruritus
HP:0000992Cutaneous photosensitivity
HP:0010472Abnormal circulating porphyrin concentration
HP:0010783Erythema
Периодический (5–29%)6
HP:0000964Eczematoid dermatitis
HP:0000969Edema
HP:0001081Cholelithiasis
HP:0001394Cirrhosis
HP:0001410Decreased liver function
HP:0001935Microcytic anemia

Эпидемиология (22)

Annual incidence
<1 / 1 000 000
Europe
Point prevalence
1-9 / 1 000 000
Europe
Annual incidence
<1 / 1 000 000
France
Point prevalence
1-9 / 1 000 000
France
Annual incidence
<1 / 1 000 000
Italy
Point prevalence
1-9 / 1 000 000
Ireland
Annual incidence
<1 / 1 000 000
Netherlands
Point prevalence
1-9 / 1 000 000
Italy
Annual incidence
<1 / 1 000 000
Norway
Point prevalence
1-9 / 100 000
Netherlands
Annual incidence
<1 / 1 000 000
Poland
Point prevalence
1-9 / 100 000
Norway
Annual incidence
<1 / 1 000 000
Spain
Point prevalence
1-9 / 1 000 000
Poland
Annual incidence
<1 / 1 000 000
Sweden
Point prevalence
1-9 / 1 000 000
Spain
Annual incidence
<1 / 1 000 000
Switzerland
Point prevalence
1-9 / 100 000
Sweden
Annual incidence
<1 / 1 000 000
United Kingdom
Point prevalence
1-9 / 100 000
Switzerland
Point prevalence
1-9 / 100 000
United Kingdom
Point prevalence
1-9 / 100 000
Slovenia

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы