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Sialuria

ORPHA:3166DiseaseAutosomal dominantInfancy

Ассоциированные гены (1)

GNE
glucosamine (UDP-N-acetyl)-2-epimerase/N-acetylmannosamine kinase
Disease-causing germline mutation(s) in
OMIM: 603824

Фенотипы (36)

Очень частый (80–99%)35
HP:0000219Thin upper lip vermilion
HP:0000280Coarse facial features
HP:0000286Epicanthus
HP:0000316Hypertelorism
HP:0000319Smooth philtrum
HP:0000369Low-set ears
HP:0000431Wide nasal bridge
HP:0000629Periorbital fullness
HP:0000943Dysostosis multiplex
HP:0001081Cholelithiasis
HP:0001250Seizure
HP:0001256Intellectual disability, mild
HP:0001290Generalized hypotonia
HP:0001382Joint hypermobility
HP:0001433Hepatosplenomegaly
HP:0001609Hoarse voice
HP:0001847Long hallux
HP:0001939Abnormality of metabolism/homeostasis
HP:0001999Abnormal facial shape
HP:0002240Hepatomegaly
HP:0002354Memory impairment
HP:0002474Expressive language delay
HP:0002487Hyperkinetic movements
HP:0002574Episodic abdominal pain
HP:0002705High, narrow palate
HP:0002781Upper airway obstruction
HP:0002910Elevated circulating hepatic transaminase concentration
HP:0003645Prolonged partial thromboplastin time
HP:00046912-3 toe syndactyly
HP:0007018Attention deficit hyperactivity disorder
HP:0008151Prolonged prothrombin time
HP:0008443Spinal deformities
HP:0010535Sleep apnea
HP:0011220Prominent forehead
HP:0012103Abnormality of the mitochondrion
Периодический (5–29%)1
HP:0001263Global developmental delay

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