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Young-onset Parkinson disease

ORPHA:2828DiseaseAutosomal recessiveAdult

Ассоциированные гены (11)

SYNJ1
synaptojanin 1
Disease-causing germline mutation(s) in
OMIM: 604297
SNCA
synuclein alpha
Candidate gene tested in
OMIM: 163890
PINK1
PTEN induced kinase 1
Disease-causing germline mutation(s) in
OMIM: 608309
UCHL1
ubiquitin C-terminal hydrolase L1
Candidate gene tested in
OMIM: 191342
HTRA2
HtrA serine peptidase 2
Candidate gene tested in
OMIM: 606441
PODXL
podocalyxin like
Disease-causing germline mutation(s) (loss of function) in
OMIM: 602632
LRRK2
leucine rich repeat kinase 2
Candidate gene tested in
OMIM: 609007
PRKN
parkin RBR E3 ubiquitin protein ligase
Disease-causing germline mutation(s) in
OMIM: 602544
PARK7
Parkinsonism associated deglycase
Disease-causing germline mutation(s) in
OMIM: 602533
VPS13C
vacuolar protein sorting 13 homolog C
Disease-causing germline mutation(s) (loss of function) in
OMIM: 608879
DNAJC6
DnaJ heat shock protein family (Hsp40) member C6
Disease-causing germline mutation(s) (loss of function) in
OMIM: 608375

Фенотипы (34)

Очень частый (80–99%)1
HP:0002063Rigidity
Частый (30–79%)6
HP:0000716Depression
HP:0000738Hallucinations
HP:0000741Apathy
HP:0001337Tremor
HP:0002172Postural instability
HP:0100660Dyskinesia
Периодический (5–29%)23
HP:0012760Reduced social responsiveness
HP:0000551Color vision defect
HP:0000726Dementia
HP:0000736Short attention span
HP:0000739Anxiety
HP:0001257Spasticity
HP:0001332Dystonia
HP:0001347Hyperreflexia
HP:0002014Diarrhea
HP:0002018Nausea
HP:0002019Constipation
HP:0002067Bradykinesia
HP:0002141Gait imbalance
HP:0002578Gastroparesis
HP:0003394Muscle spasm
HP:0004409Hyposmia
HP:0012332Abnormal autonomic nervous system physiology
HP:0012452Restless legs
HP:0025269Panic attack
HP:0030014Female sexual dysfunction
HP:0040307Male sexual dysfunction
HP:0100543Cognitive impairment
HP:0100785Insomnia
Очень редкий (1–4%)4
HP:0000651Diplopia
HP:0000713Agitation
HP:0000727Frontal lobe dementia
HP:0100710Impulsivity

Эпидемиология (2)

Point prevalence
1-5 / 10 000
Europe
Point prevalence
1-5 / 10 000
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы