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Congenital intrinsic factor deficiency

ORPHA:332DiseaseAutosomal recessive, Not applicableChildhood

Ассоциированные гены (1)

CBLIF
cobalamin binding intrinsic factor
Disease-causing germline mutation(s) in
OMIM: 609342

Фенотипы (17)

Облигатный (100%)1
HP:0005219Absence of intrinsic factor
Очень частый (80–99%)2
HP:0001889Megaloblastic anemia
HP:0100502Decreased circulating vitamin B12 concentration
Частый (30–79%)6
HP:0000726Dementia
HP:0001328Specific learning disability
HP:0002160Hyperhomocystinemia
HP:0002719Recurrent infections
HP:0002912Methylmalonic acidemia
HP:0025406Asthenia
Периодический (5–29%)7
HP:0200143Megaloblastic erythroid hyperplasia
HP:0001510Growth delay
HP:0002315Headache
HP:0003401Paresthesia
HP:0006827Atrophy of the spinal cord
HP:0009830Peripheral neuropathy
HP:0012120Methylmalonic aciduria
Исключён (0%)1
HP:6000344Anti-intrinsic factor antibody positivity

Эпидемиология (2)

Cases/families
Worldwide
Point prevalence
<1 / 1 000 000
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы