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Early-onset epileptic encephalopathy-cortical blindness-intellectual disability-facial dysmorphism syndrome

ORPHA:411986Malformation syndromeAutosomal recessiveInfancy

Ассоциированные гены (1)

DOCK7
dedicator of cytokinesis 7
Disease-causing germline mutation(s) (loss of function) in
OMIM: 615730

Фенотипы (36)

Частый (30–79%)14
HP:0000294Low anterior hairline
HP:0000455Broad nasal tip
HP:0000463Anteverted nares
HP:0000506Telecanthus
HP:0000629Periorbital fullness
HP:0000817Reduced eye contact
HP:0001249Intellectual disability
HP:0002079Hypoplasia of the corpus callosum
HP:0002465Poor speech
HP:0010818Generalized tonic seizure
HP:0010841Multifocal epileptiform discharges
HP:0012105Occipital cortical atrophy
HP:0012110Hypoplasia of the pons
HP:0100704Cerebral visual impairment
Периодический (5–29%)22
HP:0000232Everted lower lip vermilion
HP:0000322Short philtrum
HP:0000341Narrow forehead
HP:0000414Bulbous nose
HP:0000426Prominent nasal bridge
HP:0000527Long eyelashes
HP:0000528Anophthalmia
HP:0000574Thick eyebrow
HP:0000664Synophrys
HP:0000733Abnormal repetitive mannerisms
HP:0001252Hypotonia
HP:0001336Myoclonus
HP:0002121Generalized non-motor (absence) seizure
HP:0002384Focal impaired awareness seizure
HP:0002521Hypsarrhythmia
HP:0002540Inability to walk
HP:0009748Large earlobe
HP:0009904Prominent ear helix
HP:0010819Atonic seizure
HP:0012469Infantile spasms
HP:0012471Thick vermilion border
HP:0040159Abnormal spaced incisors

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