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Juvenile Huntington disease

ORPHA:248111DiseaseAutosomal dominantAdolescent, Childhood

Ассоциированные гены (1)

HTT
huntingtin
Disease-causing germline mutation(s) in
OMIM: 613004

Фенотипы (24)

Частый (30–79%)19
HP:0000708Atypical behavior
HP:0000716Depression
HP:0000726Dementia
HP:0000737Irritability
HP:0000752Hyperactivity
HP:0001250Seizure
HP:0001251Ataxia
HP:0001332Dystonia
HP:0001347Hyperreflexia
HP:0001824Weight loss
HP:0002063Rigidity
HP:0002066Gait ataxia
HP:0002067Bradykinesia
HP:0002072Chorea
HP:0002136Broad-based gait
HP:0002500Abnormal cerebral white matter morphology
HP:0012547Abnormal involuntary eye movements
HP:0030190Oral motor hypotonia
HP:0200147Neuronal loss in basal ganglia
Периодический (5–29%)5
HP:0001272Cerebellar atrophy
HP:0001336Myoclonus
HP:0002073Progressive cerebellar ataxia
HP:0002119Ventriculomegaly
HP:0006855Cerebellar vermis atrophy

Эпидемиология (2)

Annual incidence
<1 / 1 000 000
Europe
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