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Acromesomelic dysplasia, Hunter-Thompson type

ORPHA:968Malformation syndromeAutosomal recessiveAntenatal, Neonatal

Ассоциированные гены (1)

GDF5
growth differentiation factor 5
Disease-causing germline mutation(s) (loss of function) in
OMIM: 601146

Фенотипы (18)

Очень частый (80–99%)9
HP:0001156Brachydactyly
HP:0002167Abnormality of speech or vocalization
HP:0003028Abnormality of the ankles
HP:0003042Elbow dislocation
HP:0003086Acromesomelia
HP:0007598Bilateral single transverse palmar creases
HP:0008368Tarsal synostosis
HP:0008890Severe short-limb dwarfism
HP:0009778Short thumb
Частый (30–79%)9
HP:0001387Joint stiffness
HP:0002644Abnormality of pelvic girdle bone morphology
HP:0002650Scoliosis
HP:0002827Hip dislocation
HP:0002999Patellar dislocation
HP:0006011Cuboidal metacarpal
HP:0006014Abnormally shaped carpal bones
HP:0010049Short metacarpal
HP:0100543Cognitive impairment

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