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Achondroplasia

ORPHA:15DiseaseAutosomal dominantAntenatal, Neonatal

Ассоциированные гены (1)

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

Фенотипы (39)

Очень частый (80–99%)5
HP:0002808Kyphosis
HP:0002979Bowing of the legs
HP:0003498Disproportionate short stature
HP:0005619Thoracolumbar kyphosis
HP:0009826Limb undergrowth
Частый (30–79%)23
HP:0000242Parietal bossing
HP:0000256Macrocephaly
HP:0000309Abnormal midface morphology
HP:0000365Hearing impairment
HP:0000463Anteverted nares
HP:0001156Brachydactyly
HP:0001377Limited elbow extension
HP:0002007Frontal bossing
HP:0002870Obstructive sleep apnea
HP:0002938Lumbar hyperlordosis
HP:0003026Short long bone
HP:0003194Short nasal bridge
HP:0003416Spinal canal stenosis
HP:0004060Trident hand
HP:0005280Depressed nasal bridge
HP:0005819Short middle phalanx of finger
HP:0008445Cervical spinal canal stenosis
HP:0008947Floppy infant
HP:0010241Short proximal phalanx of finger
HP:0010536Central sleep apnea
HP:0011452Functional abnormality of the middle ear
HP:0045086Knee joint hypermobility
HP:0045087Hip joint hypermobility
Периодический (5–29%)10
HP:0000260Wide anterior fontanel
HP:0000956Acanthosis nigricans
HP:0001513Obesity
HP:0002091Restrictive ventilatory defect
HP:0003180Flat acetabular roof
HP:0003375Narrow greater sciatic notch
HP:0005257Thoracic hypoplasia
HP:0008905Rhizomelia
HP:0011867Abnormality of the wing of the ilium
HP:0012418Hypoxemia
Очень редкий (1–4%)1
HP:0000238Hydrocephalus

Эпидемиология (31)

Prevalence at birth
1-9 / 100 000
Worldwide
Prevalence at birth
1-9 / 100 000
Europe
Prevalence at birth
1-9 / 100 000
France
Prevalence at birth
1-9 / 100 000
Italy
Prevalence at birth
1-9 / 100 000
Sweden
Prevalence at birth
1-9 / 100 000
Spain
Prevalence at birth
1-9 / 100 000
Denmark
Prevalence at birth
1-9 / 100 000
Latin America
Prevalence at birth
1-9 / 100 000
Australia
Prevalence at birth
1-9 / 100 000
Switzerland
Prevalence at birth
1-9 / 100 000
United Kingdom
Prevalence at birth
1-9 / 100 000
Ukraine
Prevalence at birth
6-9 / 10 000
Iraq
Prevalence at birth
1-5 / 10 000
Iran, Islamic Republic of
Prevalence at birth
1-5 / 10 000
Kuwait
Prevalence at birth
1-5 / 10 000
Lebanon
Prevalence at birth
1-5 / 10 000
Saudi Arabia
Prevalence at birth
1-5 / 10 000
United Arab Emirates
Prevalence at birth
1-5 / 10 000
Cameroon
Prevalence at birth
1-9 / 100 000
North America
Prevalence at birth
1-9 / 100 000
Cuba
Prevalence at birth
1-9 / 100 000
Mexico
Prevalence at birth
1-9 / 100 000
Argentina
Prevalence at birth
1-5 / 10 000
Venezuela
Prevalence at birth
1-9 / 100 000
Germany
Prevalence at birth
1-9 / 100 000
Malta
Prevalence at birth
1-9 / 100 000
Netherlands
Prevalence at birth
1-9 / 100 000
Norway
Prevalence at birth
1-9 / 100 000
Poland
Prevalence at birth
1-5 / 10 000
Nigeria
Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы