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Dysspondyloenchondromatosis

ORPHA:85198Malformation syndromeAutosomal dominant, Not applicableInfancy, Neonatal

Ассоциированные гены (1)

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

Фенотипы (23)

Очень частый (80–99%)13
HP:0001373Joint dislocation
HP:0002650Scoliosis
HP:0002657Spondylometaphyseal dysplasia
HP:0002751Kyphoscoliosis
HP:0002761Generalized joint laxity
HP:0003037Enlarged joints
HP:0003422Vertebral segmentation defect
HP:0004322Short stature
HP:0005701Multiple enchondromatosis
HP:0012221Pretibial blistering
HP:0100559Lower limb asymmetry
HP:0100777Exostoses
HP:0200041Skin erosion
Частый (30–79%)8
HP:0000926Platyspondyly
HP:0002750Delayed skeletal maturation
HP:0002758Osteoarthritis
HP:0002857Genu valgum
HP:0002879Anisospondyly
HP:0002991Abnormal fibula morphology
HP:0004039Abnormality of ulnar metaphysis
HP:0005868Metaphyseal enchondromatosis
Периодический (5–29%)2
HP:0001249Intellectual disability
HP:0002514Cerebral calcification

Эпидемиология (3)

Cases/families
Worldwide
Point prevalence
<1 / 1 000 000
Europe
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