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Keppen-Lubinsky syndrome

ORPHA:435628Malformation syndromeAutosomal dominant, Not applicableNeonatal

Ассоциированные гены (1)

KCNJ6
potassium inwardly rectifying channel subfamily J member 6
Disease-causing germline mutation(s) in
OMIM: 600877

Фенотипы (42)

Облигатный (100%)2
HP:0005328Progeroid facial appearance
HP:0009059Congenital generalized lipodystrophy
Очень частый (80–99%)7
HP:0000194Open mouth
HP:0000252Microcephaly
HP:0000322Short philtrum
HP:0000347Micrognathia
HP:0001249Intellectual disability
HP:0001347Hyperreflexia
HP:0100678Premature skin wrinkling
Частый (30–79%)31
HP:0000212Gingival overgrowth
HP:0000218High palate
HP:0000290Abnormality of the forehead
HP:0000292Loss of facial adipose tissue
HP:0000298Mask-like facies
HP:0000430Underdeveloped nasal alae
HP:0000446Narrow nasal bridge
HP:0000496Abnormality of eye movement
HP:0000520Proptosis
HP:0000586Shallow orbits
HP:0001090Abnormally large globe
HP:0001276Hypertonia
HP:0001285Spastic tetraparesis
HP:0001371Flexion contracture
HP:0001508Failure to thrive
HP:0001561Polyhydramnios
HP:0002093Respiratory insufficiency
HP:0002094Dyspnea
HP:0002179Opisthotonus
HP:0002187Intellectual disability, profound
HP:0002650Scoliosis
HP:0002781Upper airway obstruction
HP:0005274Prominent nasal tip
HP:0006532Recurrent pneumonia
HP:0008734Decreased testicular size
HP:0008897Postnatal growth retardation
HP:0009125Lipodystrophy
HP:0009933Narrow naris
HP:0010751Chin dimple
HP:0010804Tented upper lip vermilion
HP:0011344Severe global developmental delay
Периодический (5–29%)2
HP:0001250Seizure
HP:0002659Increased susceptibility to fractures

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