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Congenital insensitivity to pain with severe intellectual disability

ORPHA:453510DiseaseAutosomal recessiveNeonatal

Ассоциированные гены (1)

CLTCL1
clathrin heavy chain like 1
Disease-causing germline mutation(s) in
OMIM: 601273

Фенотипы (29)

Частый (30–79%)11
HP:0000486Strabismus
HP:0000491Keratitis
HP:0000742Self-mutilation
HP:0001328Specific learning disability
HP:0002188Delayed CNS myelination
HP:0002754Osteomyelitis
HP:0002757Recurrent fractures
HP:0007021Pain insensitivity
HP:0008000Decreased corneal reflex
HP:0010830Impaired tactile sensation
HP:0011344Severe global developmental delay
Периодический (5–29%)18
HP:0000324Facial asymmetry
HP:0000347Micrognathia
HP:0000448Prominent nose
HP:0001518Small for gestational age
HP:0001562Oligohydramnios
HP:0001772Talipes equinovalgus
HP:0001838Rocker bottom foot
HP:0001999Abnormal facial shape
HP:0002069Bilateral tonic-clonic seizure
HP:0002982Tibial bowing
HP:0008780Congenital bilateral hip dislocation
HP:0008947Floppy infant
HP:0009826Limb undergrowth
HP:0010841Multifocal epileptiform discharges
HP:0011470Nasogastric tube feeding in infancy
HP:0012044Seesaw nystagmus
HP:0012745Short palpebral fissure
HP:0200020Corneal erosion

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