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SHORT syndrome

ORPHA:3163Malformation syndromeAutosomal dominantNeonatal

Ассоциированные гены (1)

PIK3R1
phosphoinositide-3-kinase regulatory subunit 1
Disease-causing germline mutation(s) in
OMIM: 171833

Фенотипы (37)

Очень частый (80–99%)6
HP:0001382Joint hypermobility
HP:0000023Inguinal hernia
HP:0000407Sensorineural hearing impairment
HP:0000490Deeply set eye
HP:0003510Severe short stature
HP:0007676Hypoplasia of the iris
Частый (30–79%)17
HP:0000164Abnormality of the dentition
HP:0000271Abnormality of the face
HP:0000485Megalocornea
HP:0000501Glaucoma
HP:0000615Abnormal pupil morphology
HP:0000682Abnormality of dental enamel
HP:0000691Microdontia
HP:0000819Diabetes mellitus
HP:0000855Insulin resistance
HP:0001596Alopecia
HP:0001824Weight loss
HP:0002167Abnormality of speech or vocalization
HP:0004396Poor appetite
HP:0007392Excessive wrinkled skin
HP:0008070Sparse hair
HP:0009125Lipodystrophy
HP:0011800Midface retrusion
Периодический (5–29%)14
HP:0000272Malar flattening
HP:0000277Abnormality of the mandible
HP:0000316Hypertelorism
HP:0000325Triangular face
HP:0000336Prominent supraorbital ridges
HP:0000431Wide nasal bridge
HP:0000506Telecanthus
HP:0000593Abnormal anterior chamber morphology
HP:0000627Posterior embryotoxon
HP:0001156Brachydactyly
HP:0004279Short palm
HP:0007957Corneal opacity
HP:0010668Abnormal zygomatic bone morphology
HP:0011220Prominent forehead

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