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Multiple synostoses syndrome

ORPHA:3237Malformation syndromeAutosomal dominantChildhood

Ассоциированные гены (4)

GDF5
growth differentiation factor 5
Disease-causing germline mutation(s) (gain of function) in
OMIM: 601146
NOG
noggin
Disease-causing germline mutation(s) in
OMIM: 602991
FGF9
fibroblast growth factor 9
Disease-causing germline mutation(s) in
OMIM: 600921
GDF6
growth differentiation factor 6
Disease-causing germline mutation(s) in
OMIM: 601147

Фенотипы (10)

Очень частый (80–99%)5
HP:0000405Conductive hearing impairment
HP:0001156Brachydactyly
HP:0001387Joint stiffness
HP:0004279Short palm
HP:0009773Symphalangism affecting the phalanges of the hand
Частый (30–79%)3
HP:0007598Bilateral single transverse palmar creases
HP:0010579Cone-shaped epiphysis
HP:0011304Broad thumb
Периодический (5–29%)2
HP:0000324Facial asymmetry
HP:0001597Abnormality of the nail

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