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Gerstmann-Straussler-Scheinker syndrome

ORPHA:356DiseaseAutosomal dominant, Not applicableAdult

Ассоциированные гены (1)

PRNP
prion protein (Kanno blood group)
Disease-causing germline mutation(s) in
OMIM: 176640

Фенотипы (18)

Очень частый (80–99%)4
HP:0002066Gait ataxia
HP:0007340Lower limb muscle weakness
HP:0012534Dysesthesia
HP:0100543Cognitive impairment
Частый (30–79%)13
HP:0000726Dementia
HP:0001260Dysarthria
HP:0001268Mental deterioration
HP:0001284Areflexia
HP:0001315Reduced tendon reflexes
HP:0001317Abnormal cerebellum morphology
HP:0002062Morphological abnormality of the pyramidal tract
HP:0002071Abnormality of extrapyramidal motor function
HP:0002360Sleep abnormality
HP:0003401Paresthesia
HP:0011730Abnormality of central sensory function
HP:0031006Acroparesthesia
HP:0410263Brain imaging abnormality
Периодический (5–29%)1
HP:0045084Limb myoclonus

Эпидемиология (2)

Point prevalence
Unknown
Worldwide
Annual incidence
<1 / 1 000 000
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы