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Lethal congenital contracture syndrome type 1

ORPHA:1486Malformation syndromeAutosomal recessiveAntenatal, Neonatal

Ассоциированные гены (1)

GLE1
GLE1 RNA export mediator
Disease-causing germline mutation(s) in
OMIM: 603371

Фенотипы (18)

Очень частый (80–99%)6
HP:0000316Hypertelorism
HP:0000347Micrognathia
HP:0002089Pulmonary hypoplasia
HP:0003202Skeletal muscle atrophy
HP:0003272Abnormality of the hip bone
HP:0004322Short stature
Частый (30–79%)11
HP:0000465Webbed neck
HP:0000470Short neck
HP:0000772Abnormal rib morphology
HP:0001376Limitation of joint mobility
HP:0001561Polyhydramnios
HP:0002757Recurrent fractures
HP:0003100Slender long bone
HP:0003103Abnormal cortical bone morphology
HP:0009775Amniotic constriction ring
HP:0009811Abnormality of the elbow
HP:0000358Posteriorly rotated ears
Периодический (5–29%)1
HP:0003312Abnormal form of the vertebral bodies

Эпидемиология (2)

Point prevalence
<1 / 1 000 000
Finland
Prevalence at birth
1-9 / 100 000
Finland

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы