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Acromicric dysplasia

ORPHA:969Malformation syndromeAutosomal dominantNeonatal

Ассоциированные гены (2)

FBN1
fibrillin 1
Disease-causing germline mutation(s) in
OMIM: 134797
LTBP3
latent transforming growth factor beta binding protein 3
Disease-causing germline mutation(s) in
OMIM: 602090

Фенотипы (22)

Очень частый (80–99%)10
HP:0000311Round face
HP:0000343Long philtrum
HP:0000463Anteverted nares
HP:0000527Long eyelashes
HP:0000534Abnormal eyebrow morphology
HP:0001156Brachydactyly
HP:0003196Short nose
HP:0003510Severe short stature
HP:0004279Short palm
HP:0200055Small hand
Частый (30–79%)4
HP:0000160Narrow mouth
HP:0000179Thick lower lip vermilion
HP:0000414Bulbous nose
HP:0000762Decreased nerve conduction velocity
Периодический (5–29%)8
HP:0001387Joint stiffness
HP:0001609Hoarse voice
HP:0002750Delayed skeletal maturation
HP:0002823Abnormality of femur morphology
HP:0003300Ovoid vertebral bodies
HP:0005900Fifth metacarpal with ulnar notch
HP:0005930Abnormality of epiphysis morphology
HP:0010049Short metacarpal

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