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Hypochondroplasia

ORPHA:429DiseaseAutosomal dominantAdolescent, Adult, Antenatal, Childhood, Infancy, Neonatal

Ассоциированные гены (1)

FGFR3
fibroblast growth factor receptor 3
Disease-causing germline mutation(s) (gain of function) in
OMIM: 134934

Фенотипы (20)

Очень частый (80–99%)6
HP:0001156Brachydactyly
HP:0001831Short toe
HP:0002652Skeletal dysplasia
HP:0002983Micromelia
HP:0003312Abnormal form of the vertebral bodies
HP:0011405Childhood onset short-limb short stature
Частый (30–79%)6
HP:0000944Abnormal metaphysis morphology
HP:0002644Abnormality of pelvic girdle bone morphology
HP:0002823Abnormality of femur morphology
HP:0002970Genu varum
HP:0009811Abnormality of the elbow
HP:0001382Joint hypermobility
Периодический (5–29%)8
HP:0000256Macrocephaly
HP:0001249Intellectual disability
HP:0002650Scoliosis
HP:0002758Osteoarthritis
HP:0003307Hyperlordosis
HP:0003416Spinal canal stenosis
HP:0006487Bowing of the long bones
HP:0010535Sleep apnea

Эпидемиология (2)

Prevalence at birth
1-9 / 100 000
Worldwide
Point prevalence
1-9 / 100 000
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы