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Spinocerebellar ataxia type 29

ORPHA:208513DiseaseAutosomal dominant, Autosomal recessiveInfancy, Neonatal

Ассоциированные гены (1)

ITPR1
inositol 1,4,5-trisphosphate receptor type 1
Disease-causing germline mutation(s) in
OMIM: 147265

Фенотипы (20)

Очень частый (80–99%)7
HP:0000750Delayed speech and language development
HP:0001260Dysarthria
HP:0001310Dysmetria
HP:0002066Gait ataxia
HP:0002080Intention tremor
HP:0002194Delayed gross motor development
HP:0010862Delayed fine motor development
Частый (30–79%)10
HP:0000570Abnormal saccadic eye movements
HP:0000639Nystagmus
HP:0000657Oculomotor apraxia
HP:0001270Motor delay
HP:0001272Cerebellar atrophy
HP:0001290Generalized hypotonia
HP:0002075Dysdiadochokinesis
HP:0006855Cerebellar vermis atrophy
HP:0012434Delayed social development
HP:0100543Cognitive impairment
Периодический (5–29%)3
HP:0001251Ataxia
HP:0001263Global developmental delay
HP:0025405Visual fixation instability

Эпидемиология (2)

Cases/families
Worldwide
Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы