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Horizontal gaze palsy with progressive scoliosis

ORPHA:2744DiseaseAutosomal recessiveAdolescent, Childhood

Ассоциированные гены (2)

DCC
DCC netrin 1 receptor
Disease-causing germline mutation(s) in
OMIM: 120470
ROBO3
roundabout guidance receptor 3
Disease-causing germline mutation(s) in
OMIM: 608630

Фенотипы (16)

Очень частый (80–99%)3
HP:0002650Scoliosis
HP:0002808Kyphosis
HP:0007817Horizontal supranuclear gaze palsy
Частый (30–79%)11
HP:0000252Microcephaly
HP:0000470Short neck
HP:0000486Strabismus
HP:0000505Visual impairment
HP:0000639Nystagmus
HP:0001252Hypotonia
HP:0002750Delayed skeletal maturation
HP:0003198Myopathy
HP:0003508Proportionate short stature
HP:0009921Duane anomaly
HP:0100543Cognitive impairment
Периодический (5–29%)2
HP:0000407Sensorineural hearing impairment
HP:0001250Seizure

Эпидемиология (1)

Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы