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Spinocerebellar ataxia type 36

ORPHA:276198DiseaseAutosomal dominantAdult

Ассоциированные гены (1)

NOP56
NOP56 ribonucleoprotein
Disease-causing germline mutation(s) in
OMIM: 614154

Фенотипы (27)

Очень частый (80–99%)5
HP:0000365Hearing impairment
HP:0001251Ataxia
HP:0001260Dysarthria
HP:0002070Limb ataxia
HP:0002078Truncal ataxia
Частый (30–79%)10
HP:0000514Slow saccadic eye movements
HP:0000622Blurred vision
HP:0001288Gait disturbance
HP:0001308Tongue fasciculations
HP:0001310Dysmetria
HP:0002380Fasciculations
HP:0003202Skeletal muscle atrophy
HP:0003487Babinski sign
HP:0007001Loss of Purkinje cells in the cerebellar vermis
HP:0012473Tongue atrophy
Периодический (5–29%)2
HP:0000508Ptosis
HP:0001347Hyperreflexia
Очень редкий (1–4%)10
HP:0000651Diplopia
HP:0002015Dysphagia
HP:0002076Migraine
HP:0002080Intention tremor
HP:0002321Vertigo
HP:0002346Head tremor
HP:0002378Hand tremor
HP:0002607Bowel incontinence
HP:0007018Attention deficit hyperactivity disorder
HP:0045084Limb myoclonus

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