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Epilepsy of infancy with migrating focal seizures

ORPHA:293181DiseaseAutosomal dominant, Autosomal recessive, X-linked recessiveInfancy, Neonatal

Ассоциированные гены (9)

SCN2A
sodium voltage-gated channel alpha subunit 2
Disease-causing germline mutation(s) in
OMIM: 182390
KCNQ2
potassium voltage-gated channel subfamily Q member 2
Disease-causing germline mutation(s) in
OMIM: 602235
PIGA
phosphatidylinositol glycan anchor biosynthesis class A
Disease-causing germline mutation(s) in
OMIM: 311770
SCN1A
sodium voltage-gated channel alpha subunit 1
Disease-causing germline mutation(s) in
OMIM: 182389
SLC25A22
solute carrier family 25 member 22
Disease-causing germline mutation(s) in
OMIM: 609302
TBC1D24
TBC1 domain family member 24
Disease-causing germline mutation(s) (loss of function) in
OMIM: 613577
PLCB1
phospholipase C beta 1
Disease-causing germline mutation(s) in
OMIM: 607120
KCNT1
potassium sodium-activated channel subfamily T member 1
Disease-causing germline mutation(s) (gain of function) in
OMIM: 608167
SLC12A5
solute carrier family 12 member 5
Disease-causing germline mutation(s) (loss of function) in
OMIM: 606726

Фенотипы (25)

Очень частый (80–99%)6
HP:0002376Developmental regression
HP:0002540Inability to walk
HP:0004302Functional motor deficit
HP:0010841Multifocal epileptiform discharges
HP:0012758Neurodevelopmental delay
HP:0100543Cognitive impairment
Частый (30–79%)9
HP:0000252Microcephaly
HP:0001252Hypotonia
HP:0002059Cerebral atrophy
HP:0002069Bilateral tonic-clonic seizure
HP:0002384Focal impaired awareness seizure
HP:0006813Focal hemiclonic seizure
HP:0007334Bilateral tonic-clonic seizure with focal onset
HP:0010821Focal emotional seizure with laughing
HP:0032794Myoclonic seizure
Периодический (5–29%)9
HP:0000505Visual impairment
HP:0001273Abnormal corpus callosum morphology
HP:0001276Hypertonia
HP:0001508Failure to thrive
HP:0002521Hypsarrhythmia
HP:0002650Scoliosis
HP:0011097Epileptic spasm
HP:0012448Delayed myelination
HP:0031834Aortopulmonary collateral arteries
Очень редкий (1–4%)1
HP:0000826Precocious puberty

Эпидемиология (4)

Cases/families
Worldwide
Point prevalence
<1 / 1 000 000
Worldwide
Point prevalence
1-9 / 1 000 000
United Kingdom
Prevalence at birth
1-9 / 1 000 000
United Kingdom

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы