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Imerslund-Gräsbeck syndrome

ORPHA:35858DiseaseAutosomal recessiveChildhood

Ассоциированные гены (2)

AMN
amnion associated transmembrane protein
Disease-causing germline mutation(s) in
OMIM: 605799
CUBN
cubilin
Disease-causing germline mutation(s) in
OMIM: 602997

Фенотипы (30)

Облигатный (100%)3
HP:0100502Decreased circulating vitamin B12 concentration
HP:0200118Malabsorption of Vitamin B12
HP:0410216Abnormal blood 5-methyltetrahydrofolate level
Очень частый (80–99%)3
HP:0001889Megaloblastic anemia
HP:0001972Macrocytic anemia
HP:0004821Hypersegmentation of neutrophil nuclei
Частый (30–79%)7
HP:0000093Proteinuria
HP:0000980Pallor
HP:0001875Decreased total neutrophil count
HP:0001923Reticulocytosis
HP:0004823Anisopoikilocytosis
HP:0020061Abnormal hemoglobin concentration
HP:0032566Oval macrocytosis
Периодический (5–29%)16
HP:0000750Delayed speech and language development
HP:0001252Hypotonia
HP:0001508Failure to thrive
HP:0001649Tachycardia
HP:0001824Weight loss
HP:0001873Thrombocytopenia
HP:0001876Pancytopenia
HP:0001892Abnormal bleeding
HP:0002013Vomiting
HP:0000206Glossitis
HP:0000707Abnormality of the nervous system
HP:0002019Constipation
HP:0002376Developmental regression
HP:0004396Poor appetite
HP:0030318Angular cheilitis
HP:0031936Delayed ability to walk
Очень редкий (1–4%)1
HP:0002721Immunodeficiency

Эпидемиология (4)

Point prevalence
1-9 / 1 000 000
Finland
Point prevalence
1-9 / 1 000 000
Norway
Point prevalence
Unknown
Worldwide
Point prevalence
1-9 / 1 000 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы