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Atelosteogenesis type III

ORPHA:56305Malformation syndromeAutosomal dominant, Not applicableAntenatal, Infancy, Neonatal

Ассоциированные гены (1)

FLNB
filamin B
Disease-causing germline mutation(s) in
OMIM: 603381

Фенотипы (27)

Частый (30–79%)11
HP:0031095Abnormal humerus morphology
HP:0001248Short tubular bones of the hand
HP:0001762Talipes equinovarus
HP:0002093Respiratory insufficiency
HP:0002827Hip dislocation
HP:0002999Patellar dislocation
HP:0003042Elbow dislocation
HP:0003417Coronal cleft vertebrae
HP:0004976Knee dislocation
HP:0006408Distal tapering femur
HP:0008417Vertebral hypoplasia
Периодический (5–29%)16
HP:0000218High palate
HP:0000347Micrognathia
HP:0001188Hand clenching
HP:0001263Global developmental delay
HP:0001561Polyhydramnios
HP:0002990Fibular aplasia
HP:0003049Ulnar deviation of the wrist
HP:0003862Absent humerus
HP:0003902Epiphyseal stippling of the humerus
HP:0003974Absent radius
HP:0005257Thoracic hypoplasia
HP:0005619Thoracolumbar kyphosis
HP:0005736Short tibia
HP:0005905Abnormal cervical curvature
HP:0006384Club-shaped distal femur
HP:0008755Laryngotracheomalacia

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