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Dentatorubral pallidoluysian atrophy

ORPHA:101DiseaseAutosomal dominantAll ages

Ассоциированные гены (1)

ATN1
atrophin 1
Disease-causing germline mutation(s) in
OMIM: 607462

Фенотипы (26)

Очень частый (80–99%)1
HP:0002073Progressive cerebellar ataxia
Частый (30–79%)22
HP:0002075Dysdiadochokinesis
HP:0000597Ophthalmoparesis
HP:0000639Nystagmus
HP:0000726Dementia
HP:0001138Optic neuropathy
HP:0001152Saccadic smooth pursuit
HP:0001250Seizure
HP:0001251Ataxia
HP:0001260Dysarthria
HP:0001265Hyporeflexia
HP:0001266Choreoathetosis
HP:0001310Dysmetria
HP:0001336Myoclonus
HP:0002066Gait ataxia
HP:0002070Limb ataxia
HP:0002078Truncal ataxia
HP:0002345Action tremor
HP:0004305Involuntary movements
HP:0010831Impaired proprioception
HP:0010867Dyssynergia
HP:0030890Hyperintensity of cerebral white matter on MRI
HP:0100543Cognitive impairment
Периодический (5–29%)3
HP:0000643Blepharospasm
HP:0002354Memory impairment
HP:0012048Oromandibular dystonia

Эпидемиология (3)

Point prevalence
1-9 / 1 000 000
Japan
Point prevalence
1-9 / 1 000 000
United Kingdom
Point prevalence
1-9 / 1 000 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы