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Bilateral perisylvian polymicrogyria

ORPHA:98889Clinical subtypeAutosomal recessiveAntenatal, Infancy, Neonatal

Ассоциированные гены (3)

SRPX2
sushi repeat containing protein X-linked 2
Candidate gene tested in
OMIM: 300642
ADGRG1
adhesion G protein-coupled receptor G1
Disease-causing germline mutation(s) in
OMIM: 604110
PI4KA
phosphatidylinositol 4-kinase alpha
Disease-causing germline mutation(s) in
OMIM: 600286

Фенотипы (49)

Очень частый (80–99%)2
HP:0002269Abnormality of neuronal migration
HP:0032407Bilateral perisylvian polymicrogyria
Частый (30–79%)17
HP:0000750Delayed speech and language development
HP:0001249Intellectual disability
HP:0001250Seizure
HP:0001257Spasticity
HP:0001263Global developmental delay
HP:0001328Specific learning disability
HP:0001347Hyperreflexia
HP:0001371Flexion contracture
HP:0002392EEG with polyspike wave complexes
HP:0002463Language impairment
HP:0005684Distal arthrogryposis
HP:0007359Focal-onset seizure
HP:0008947Floppy infant
HP:0012014EEG with central focal spikes
HP:0012017EEG with parietal focal spikes
HP:0020190Perisylvian predominant thick cortex pachygyria
HP:0030319Weakness of facial musculature
Периодический (5–29%)30
HP:0000252Microcephaly
HP:0000347Micrognathia
HP:0000365Hearing impairment
HP:0000453Choanal atresia
HP:0000639Nystagmus
HP:0000767Pectus excavatum
HP:0001260Dysarthria
HP:0001310Dysmetria
HP:0001320Cerebellar vermis hypoplasia
HP:0001349Facial diplegia
HP:0001511Intrauterine growth retardation
HP:0002015Dysphagia
HP:0002020Gastroesophageal reflux
HP:0002061Lower limb spasticity
HP:0002104Apnea
HP:0002307Drooling
HP:0002385Paraparesis
HP:0002509Limb hypertonia
HP:0002510Spastic tetraplegia
HP:0002835Aspiration
HP:0007024Pseudobulbar paralysis
HP:0007033Cerebellar dysplasia
HP:0007301Oromotor apraxia
HP:0010808Protruding tongue
HP:0011157Focal sensory seizure
HP:0011755Ectopic posterior pituitary
HP:0011968Feeding difficulties
HP:0012015EEG with frontal focal spikes
HP:0012469Infantile spasms
HP:0410011Abnormality of masticatory muscle

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы