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Acromesomelic dysplasia, Grebe type

ORPHA:2098Malformation syndromeAutosomal recessiveAntenatal, Neonatal

Ассоциированные гены (2)

BMPR1B
bone morphogenetic protein receptor type 1B
Disease-causing germline mutation(s) (loss of function) in
OMIM: 603248
GDF5
growth differentiation factor 5
Disease-causing germline mutation(s) (loss of function) in
OMIM: 601146

Фенотипы (18)

Очень частый (80–99%)13
HP:0001156Brachydactyly
HP:0001387Joint stiffness
HP:0001773Short foot
HP:0001831Short toe
HP:0002652Skeletal dysplasia
HP:0002983Micromelia
HP:0005048Synostosis of carpal bones
HP:0005914Aplasia/Hypoplasia involving the metacarpal bones
HP:0006487Bowing of the long bones
HP:0008368Tarsal synostosis
HP:0008873Disproportionate short-limb short stature
HP:0100242Sarcoma
HP:0100387Aplasia of the middle phalanges of the toes
Частый (30–79%)4
HP:0001162Postaxial hand polydactyly
HP:0003038Fibular hypoplasia
HP:0005736Short tibia
HP:0009601Aplasia/Hypoplasia of the thumb
Периодический (5–29%)1
HP:0001522Death in infancy

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