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Spinocerebellar ataxia type 2

ORPHA:98756DiseaseAutosomal dominantAll ages

Ассоциированные гены (1)

ATXN2
ataxin 2
Disease-causing germline mutation(s) in
OMIM: 601517

Фенотипы (27)

Очень частый (80–99%)2
HP:0002073Progressive cerebellar ataxia
HP:0045007Abnormality of the substantia nigra
Частый (30–79%)19
HP:0000514Slow saccadic eye movements
HP:0000623Supranuclear ophthalmoplegia
HP:0000639Nystagmus
HP:0000726Dementia
HP:0001260Dysarthria
HP:0001265Hyporeflexia
HP:0001290Generalized hypotonia
HP:0001332Dystonia
HP:0002066Gait ataxia
HP:0002072Chorea
HP:0002174Postural tremor
HP:0002380Fasciculations
HP:0003133Abnormality of the spinocerebellar tracts
HP:0003394Muscle spasm
HP:0006955Olivopontocerebellar hypoplasia
HP:0008311Spinal cord posterior columns myelin loss
HP:0012082Cerebellar Purkinje layer atrophy
HP:0025461Abnormal cell morphology
HP:0030186Kinetic tremor
Периодический (5–29%)6
HP:0000597Ophthalmoparesis
HP:0001300Parkinsonism
HP:0002120Cerebral cortical atrophy
HP:0002536Abnormal cortical gyration
HP:0006801Hyperactive deep tendon reflexes
HP:0012762Cerebral white matter atrophy

Эпидемиология (1)

Point prevalence
1-9 / 100 000
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы