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Spondylocarpotarsal synostosis

ORPHA:3275Malformation syndromeAutosomal recessiveChildhood, Infancy

Ассоциированные гены (2)

MYH3
myosin heavy chain 3
Disease-causing germline mutation(s) in
OMIM: 160720
FLNB
filamin B
Disease-causing germline mutation(s) (loss of function) in
OMIM: 603381

Фенотипы (17)

Частый (30–79%)11
HP:0000365Hearing impairment
HP:0000768Pectus carinatum
HP:0001762Talipes equinovarus
HP:0002650Scoliosis
HP:0002938Lumbar hyperlordosis
HP:0002945Intervertebral space narrowing
HP:0002948Vertebral fusion
HP:0003498Disproportionate short stature
HP:0006297Enamel hypoplasia
HP:0008368Tarsal synostosis
HP:0009702Carpal synostosis
Периодический (5–29%)6
HP:0000175Cleft palate
HP:0000470Short neck
HP:0001216Delayed ossification of carpal bones
HP:0001763Pes planus
HP:0002007Frontal bossing
HP:0002750Delayed skeletal maturation

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