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Malan overgrowth syndrome

ORPHA:420179Malformation syndromeAutosomal dominant, UnknownInfancy

Ассоциированные гены (1)

NFIX
nuclear factor I X
Disease-causing germline mutation(s) in
OMIM: 164005

Фенотипы (38)

Очень частый (80–99%)1
HP:0005616Accelerated skeletal maturation
Частый (30–79%)16
HP:0000098Tall stature
HP:0000256Macrocephaly
HP:0000275Narrow face
HP:0000300Oval face
HP:0000348High forehead
HP:0000486Strabismus
HP:0000494Downslanted palpebral fissures
HP:0000767Pectus excavatum
HP:0001319Neonatal hypotonia
HP:0002079Hypoplasia of the corpus callosum
HP:0002119Ventriculomegaly
HP:0002162Low posterior hairline
HP:0002342Intellectual disability, moderate
HP:0003100Slender long bone
HP:0008872Feeding difficulties in infancy
HP:0011220Prominent forehead
Периодический (5–29%)21
HP:0000160Narrow mouth
HP:0000218High palate
HP:0000307Pointed chin
HP:0000324Facial asymmetry
HP:0000490Deeply set eye
HP:0000543Optic disc pallor
HP:0000639Nystagmus
HP:0000739Anxiety
HP:0001250Seizure
HP:0001256Intellectual disability, mild
HP:0001357Plagiocephaly
HP:0002007Frontal bossing
HP:0002076Migraine
HP:0002131Episodic ataxia
HP:0002365Hypoplasia of the brainstem
HP:0002650Scoliosis
HP:0005280Depressed nasal bridge
HP:0006956Dilation of lateral ventricles
HP:0007766Optic disc hypoplasia
HP:0010864Intellectual disability, severe
HP:0030799Scaphocephaly

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