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Spinocerebellar ataxia type 17

ORPHA:98759DiseaseAutosomal dominantAll ages

Ассоциированные гены (1)

TBP
TATA-box binding protein
Disease-causing germline mutation(s) in
OMIM: 600075

Фенотипы (19)

Очень частый (80–99%)2
HP:0001251Ataxia
HP:0001288Gait disturbance
Частый (30–79%)17
HP:0000473Torticollis
HP:0000643Blepharospasm
HP:0000708Atypical behavior
HP:0001257Spasticity
HP:0001268Mental deterioration
HP:0001272Cerebellar atrophy
HP:0001300Parkinsonism
HP:0001332Dystonia
HP:0002063Rigidity
HP:0002072Chorea
HP:0002529Neuronal loss in central nervous system
HP:0004305Involuntary movements
HP:0007058Generalized cerebral atrophy/hypoplasia
HP:0007256Abnormal pyramidal sign
HP:0007366Atrophy/Degeneration affecting the brainstem
HP:0012082Cerebellar Purkinje layer atrophy
HP:0002356Writer's cramp

Эпидемиология (4)

Cases/families
Worldwide
Point prevalence
<1 / 1 000 000
Worldwide
Point prevalence
<1 / 1 000 000
Japan
Point prevalence
1-9 / 1 000 000
United Kingdom

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы