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Sclerosteosis

ORPHA:3152Malformation syndromeAutosomal recessiveChildhood, Neonatal

Ассоциированные гены (2)

SOST
sclerostin
Disease-causing germline mutation(s) (loss of function) in
OMIM: 605740
LRP4
LDL receptor related protein 4
Disease-causing germline mutation(s) in
OMIM: 604270

Фенотипы (14)

Очень частый (80–99%)10
HP:0000098Tall stature
HP:0000366Abnormality of the nose
HP:00012332-3 finger syndactyly
HP:0003103Abnormal cortical bone morphology
HP:0004493Craniofacial hyperostosis
HP:0005019Diaphyseal thickening
HP:0006101Finger syndactyly
HP:0009838Curved distal phalanges of the hand
HP:0011001Increased bone mineral density
HP:0100798Fingernail dysplasia
Частый (30–79%)3
HP:0000407Sensorineural hearing impairment
HP:0000508Ptosis
HP:0010628Facial palsy
Периодический (5–29%)1
HP:0000648Optic atrophy

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