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Hydranencephaly

ORPHA:2177Malformation syndromeAutosomal recessive, UnknownAntenatal, Neonatal

Ассоциированные гены (1)

NDE1
nudE neurodevelopment protein 1
Disease-causing germline mutation(s) in
OMIM: 609449

Фенотипы (28)

Очень частый (80–99%)7
HP:0000618Blindness
HP:0001263Global developmental delay
HP:0001511Intrauterine growth retardation
HP:0002120Cerebral cortical atrophy
HP:0008610Infantile sensorineural hearing impairment
HP:0008897Postnatal growth retardation
HP:0010994Abnormal corpus striatum morphology
Частый (30–79%)17
HP:0000601Hypotelorism
HP:0001250Seizure
HP:0001254Lethargy
HP:0001264Spastic diplegia
HP:0001287Meningitis
HP:0002179Opisthotonus
HP:0006698Dilatation of the ventricular cavity
HP:0007023Antenatal intracerebral hemorrhage
HP:0009145Abnormal cerebral artery morphology
HP:0010652Abnormal dura mater morphology
HP:0011328Abnormality of fontanelles
HP:0025040Thalamic edema
HP:0025099Dysgenesis of the thalamus
HP:0025258Stiff neck
HP:0025517Hypoplastic hippocampus
HP:0410279Atrophic pituitary gland
HP:3000062Abnormal internal carotid artery morphology
Периодический (5–29%)4
HP:0000533Chorioretinal atrophy
HP:0000609Optic nerve hypoplasia
HP:0002119Ventriculomegaly
HP:0011451Congenital microcephaly

Эпидемиология (4)

Prevalence at birth
1-9 / 100 000
Worldwide
Point prevalence
Unknown
Worldwide
Prevalence at birth
1-9 / 100 000
Japan
Prevalence at birth
1-9 / 100 000
United States

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы