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Lennox-Gastaut syndrome

ORPHA:2382DiseaseAutosomal dominant, Multigenic/multifactorial, Not applicableChildhood, Infancy

Ассоциированные гены (8)

CUX2
cut like homeobox 2
Disease-causing germline mutation(s) in
OMIM: 610648
SCN1A
sodium voltage-gated channel alpha subunit 1
Disease-causing germline mutation(s) in
OMIM: 182389
MAPK10
mitogen-activated protein kinase 10
Candidate gene tested in
OMIM: 602897
CHD2
chromodomain helicase DNA binding protein 2
Disease-causing germline mutation(s) in
OMIM: 602119
DNM1
dynamin 1
Disease-causing germline mutation(s) in
OMIM: 602377
GABRB3
gamma-aminobutyric acid type A receptor subunit beta3
Disease-causing germline mutation(s) in
OMIM: 137192
CACNA1A
calcium voltage-gated channel subunit alpha1 A
Disease-causing germline mutation(s) (gain of function) in
OMIM: 601011
CACNA1A
calcium voltage-gated channel subunit alpha1 A
Disease-causing germline mutation(s) (loss of function) in
OMIM: 601011

Фенотипы (25)

Очень частый (80–99%)3
HP:0001249Intellectual disability
HP:0001298Encephalopathy
HP:0011195EEG with focal sharp slow waves
Частый (30–79%)16
HP:0000708Atypical behavior
HP:0000718Aggressive behavior
HP:0000729Autistic behavior
HP:0000737Irritability
HP:0000752Hyperactivity
HP:0001268Mental deterioration
HP:0001336Myoclonus
HP:0002069Bilateral tonic-clonic seizure
HP:0002353EEG abnormality
HP:0002363Abnormal brainstem morphology
HP:0002527Falls
HP:0007270Atypical absence seizure
HP:0010818Generalized tonic seizure
HP:0010819Atonic seizure
HP:0012075Personality disorder
HP:0012758Neurodevelopmental delay
Периодический (5–29%)6
HP:0000709Psychosis
HP:0000741Apathy
HP:0002123Generalized myoclonic seizure
HP:0002321Vertigo
HP:0002376Developmental regression
HP:0007359Focal-onset seizure

Эпидемиология (2)

Annual incidence
1-9 / 1 000 000
Europe
Point prevalence
1-5 / 10 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы