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Ellis Van Creveld syndrome

ORPHA:289Malformation syndromeAutosomal recessiveAntenatal, Neonatal

Ассоциированные гены (6)

DYNC2LI1
dynein cytoplasmic 2 light intermediate chain 1
Disease-causing germline mutation(s) in
OMIM: 617083
GLI1
GLI family zinc finger 1
Disease-causing germline mutation(s) (loss of function) in
OMIM: 165220
EVC2
EvC ciliary complex subunit 2
Disease-causing germline mutation(s) in
OMIM: 607261
EVC
EvC ciliary complex subunit 1
Disease-causing germline mutation(s) in
OMIM: 604831
PRKACA
protein kinase cAMP-activated catalytic subunit alpha
Disease-causing germline mutation(s) in
OMIM: 601639
PRKACB
protein kinase cAMP-activated catalytic subunit beta
Disease-causing germline mutation(s) in
OMIM: 176892

Фенотипы (51)

Очень частый (80–99%)19
HP:0000164Abnormality of the dentition
HP:0000774Narrow chest
HP:0001161Hand polydactyly
HP:0001231Abnormal fingernail morphology
HP:0001508Failure to thrive
HP:0001595Abnormality of the hair
HP:0001597Abnormality of the nail
HP:0001654Abnormal heart valve morphology
HP:0001800Hypoplastic toenails
HP:0001829Foot polydactyly
HP:0002164Nail dysplasia
HP:0002857Genu valgum
HP:0002983Micromelia
HP:0006695Atrioventricular canal defect
HP:0008921Neonatal short-limb short stature
HP:0009882Short distal phalanx of finger
HP:0010306Short thorax
HP:0011830Abnormal oral mucosa morphology
HP:0030680Abnormal cardiovascular system morphology
Частый (30–79%)18
HP:0000028Cryptorchidism
HP:0000039Epispadias
HP:0000047Hypospadias
HP:0000069Abnormality of the ureter
HP:0000077Abnormality of the kidney
HP:0000190Abnormal oral frenulum morphology
HP:0000486Strabismus
HP:0000668Hypodontia
HP:0000691Microdontia
HP:0001241Capitate-hamate fusion
HP:0001511Intrauterine growth retardation
HP:0001629Ventricular septal defect
HP:0001631Atrial septal defect
HP:0001651Dextrocardia
HP:0001696Situs inversus totalis
HP:0002644Abnormality of pelvic girdle bone morphology
HP:0006703Aplasia/Hypoplasia of the lungs
HP:0011065Conical incisor
Периодический (5–29%)14
HP:0000008Abnormal morphology of female internal genitalia
HP:0000072Hydroureter
HP:0000233Thin vermilion border
HP:0000684Delayed eruption of teeth
HP:0000924Abnormality of the skeletal system
HP:0001249Intellectual disability
HP:0002097Emphysema
HP:0002488Acute leukemia
HP:0002750Delayed skeletal maturation
HP:0002967Cubitus valgus
HP:0005048Synostosis of carpal bones
HP:0005561Abnormality of bone marrow cell morphology
HP:0008678Renal hypoplasia/aplasia
HP:0011362Abnormal hair quantity

Эпидемиология (4)

Prevalence at birth
>1 / 1000
Specific population
Prevalence at birth
1-9 / 1 000 000
Europe
Prevalence at birth
1-9 / 100 000
Worldwide
Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы