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Hereditary late-onset Parkinson disease

ORPHA:411602DiseaseAutosomal dominantAdult, Elderly

Ассоциированные гены (7)

SNCA
synuclein alpha
Disease-causing germline mutation(s) (gain of function) in
OMIM: 163890
GBA1
glucosylceramidase beta 1
Major susceptibility factor in
OMIM: 606463
LRRK2
leucine rich repeat kinase 2
Disease-causing germline mutation(s) (gain of function) in
OMIM: 609007
EIF4G1
eukaryotic translation initiation factor 4 gamma 1
Candidate gene tested in
OMIM: 600495
VPS35
VPS35 retromer complex component
Disease-causing germline mutation(s) in
OMIM: 601501
DNAJC13
DnaJ heat shock protein family (Hsp40) member C13
Disease-causing germline mutation(s) (gain of function) in
OMIM: 614334
GIGYF2
GRB10 interacting GYF protein 2
Candidate gene tested in
OMIM: 612003

Фенотипы (34)

Облигатный (100%)1
HP:0001300Parkinsonism
Частый (30–79%)9
HP:0000651Diplopia
HP:0002015Dysphagia
HP:0002304Akinesia
HP:0002322Resting tremor
HP:0002359Frequent falls
HP:0002548Parkinsonism with favorable response to dopaminergic medication
HP:0004409Hyposmia
HP:0005340Spastic/hyperactive bladder
HP:0012450Chronic constipation
Периодический (5–29%)22
HP:0000338Hypomimic face
HP:0000713Agitation
HP:0000716Depression
HP:0000741Apathy
HP:0000744Low frustration tolerance
HP:0001268Mental deterioration
HP:0001332Dystonia
HP:0001824Weight loss
HP:0002063Rigidity
HP:0002067Bradykinesia
HP:0002120Cerebral cortical atrophy
HP:0002171Gliosis
HP:0002172Postural instability
HP:0002360Sleep abnormality
HP:0002362Shuffling gait
HP:0002367Visual hallucinations
HP:0003394Muscle spasm
HP:0004926Orthostatic hypotension due to autonomic dysfunction
HP:0031435Monotonic speech
HP:0100315Lewy bodies
HP:0100660Dyskinesia
HP:0100710Impulsivity
Очень редкий (1–4%)2
HP:0000726Dementia
HP:0100753Schizophrenia

Эпидемиология (1)

Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы