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Kniest dysplasia

ORPHA:485DiseaseAutosomal dominantAntenatal, Infancy, Neonatal

Ассоциированные гены (1)

COL2A1
collagen type II alpha 1 chain
Disease-causing germline mutation(s) in
OMIM: 120140

Фенотипы (51)

Облигатный (100%)1
HP:0008271Abnormal cartilage collagen
Очень частый (80–99%)15
HP:0000311Round face
HP:0000520Proptosis
HP:0001367Abnormal joint morphology
HP:0001387Joint stiffness
HP:0001591Bell-shaped thorax
HP:0002663Delayed epiphyseal ossification
HP:0003037Enlarged joints
HP:0003330Abnormal bone structure
HP:0003498Disproportionate short stature
HP:0005280Depressed nasal bridge
HP:0007773Vitreoretinopathy
HP:0007964Degenerative vitreoretinopathy
HP:0011003High myopia
HP:0012069Keratan sulfate excretion in urine
HP:0012785Flexion contracture of finger
Частый (30–79%)19
HP:0000175Cleft palate
HP:0000365Hearing impairment
HP:0000541Retinal detachment
HP:0000926Platyspondyly
HP:0000947Dumbbell-shaped long bone
HP:0003016Metaphyseal widening
HP:0003026Short long bone
HP:0003040Arthropathy
HP:0003051Enlarged metaphyses
HP:0003311Hypoplasia of the odontoid process
HP:0003521Disproportionate short-trunk short stature
HP:0007992Lattice retinal degeneration
HP:0008063Aplasia/Hypoplasia of the lens
HP:0009815Aplasia/hypoplasia of the extremities
HP:0010306Short thorax
HP:0010574Abnormality of the epiphysis of the femoral head
HP:0010580Enlarged epiphyses
HP:0010646Cervical spine instability
HP:0012230Rhegmatogenous retinal detachment
Периодический (5–29%)12
HP:0000201Pierre-Robin sequence
HP:0000470Short neck
HP:0000518Cataract
HP:0001488Bilateral ptosis
HP:0002949Fused cervical vertebrae
HP:0003417Coronal cleft vertebrae
HP:0004557Anterior vertebral fusion
HP:0006375Dumbbell-shaped femur
HP:0006454Delayed patellar ossification
HP:0008422Vertebral wedging
HP:0012019Lens luxation
HP:0025474Erythematous plaque
Очень редкий (1–4%)4
HP:0000256Macrocephaly
HP:0002086Abnormality of the respiratory system
HP:0002176Spinal cord compression
HP:0008755Laryngotracheomalacia

Эпидемиология (1)

Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы