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Oculoskeletodental syndrome

ORPHA:557003DiseaseAutosomal recessiveAdolescent, Adult, Childhood

Ассоциированные гены (1)

PIK3C2A
phosphatidylinositol-4-phosphate 3-kinase catalytic subunit type 2 alpha
Disease-causing germline mutation(s) (loss of function) in
OMIM: 603601

Фенотипы (38)

Частый (30–79%)14
HP:0004322Short stature
HP:0011020Abnormality of mucopolysaccharide metabolism
HP:0012758Neurodevelopmental delay
HP:0000121Nephrocalcinosis
HP:0000164Abnormality of the dentition
HP:0000280Coarse facial features
HP:0000365Hearing impairment
HP:0000501Glaucoma
HP:0000519Developmental cataract
HP:0000599Abnormality of the frontal hairline
HP:0001297Stroke
HP:0001328Specific learning disability
HP:0001999Abnormal facial shape
HP:0003072Hypercalcemia
Периодический (5–29%)24
HP:0000278Retrognathia
HP:0000286Epicanthus
HP:0000405Conductive hearing impairment
HP:0000407Sensorineural hearing impairment
HP:0000431Wide nasal bridge
HP:0000677Oligodontia
HP:0000691Microdontia
HP:0002300Mutism
HP:0002650Scoliosis
HP:0002750Delayed skeletal maturation
HP:0002901Hypocalcemia
HP:0002942Thoracic kyphosis
HP:0003090Hypoplasia of the capital femoral epiphysis
HP:0003307Hyperlordosis
HP:0005280Depressed nasal bridge
HP:0006297Enamel hypoplasia
HP:0006989Dysplastic corpus callosum
HP:0007042Focal white matter lesions
HP:0009237Short 5th finger
HP:0009928Thick nasal alae
HP:0010663Abnormality of thalamus morphology
HP:0010761Broad columella
HP:0030084Clinodactyly
HP:0100255Metaphyseal dysplasia

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