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Familial focal epilepsy with variable foci

ORPHA:98820DiseaseAutosomal dominantAdolescent, Adult, Childhood, Infancy

Ассоциированные гены (4)

DEPDC5
DEP domain containing 5, GATOR1 subcomplex subunit
Disease-causing germline mutation(s) (loss of function) in
OMIM: 614191
NPRL2
NPR2 like, GATOR1 complex subunit
Disease-causing germline mutation(s) (loss of function) in
OMIM: 607072
NPRL3
NPR3 like, GATOR1 complex subunit
Disease-causing germline mutation(s) (loss of function) in
OMIM: 600928
SCN3A
sodium voltage-gated channel alpha subunit 3
Disease-causing germline mutation(s) in
OMIM: 182391

Фенотипы (26)

Очень частый (80–99%)2
HP:0007359Focal-onset seizure
HP:0011185EEG with focal epileptiform discharges
Частый (30–79%)3
HP:0002069Bilateral tonic-clonic seizure
HP:0025373Interictal EEG abnormality
HP:0031951Nocturnal seizures
Периодический (5–29%)17
HP:0000708Atypical behavior
HP:0000729Autistic behavior
HP:0000980Pallor
HP:0001249Intellectual disability
HP:0002349Focal aware seizure
HP:0002367Visual hallucinations
HP:0002384Focal impaired awareness seizure
HP:0002427Motor aphasia
HP:0003401Paresthesia
HP:0008765Auditory hallucinations
HP:0010841Multifocal epileptiform discharges
HP:0012005Deja vu
HP:0012469Infantile spasms
HP:0012531Pain
HP:0031284Flushing
HP:0032046Focal cortical dysplasia
HP:0100543Cognitive impairment
Очень редкий (1–4%)4
HP:0002126Polymicrogyria
HP:0002521Hypsarrhythmia
HP:0007206Hemimegalencephaly
HP:0011171Simple febrile seizures

Эпидемиология (2)

Cases/families
Worldwide
Point prevalence
<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