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Spondylometaphyseal dysplasia, Sedaghatian type

ORPHA:93317Malformation syndromeAutosomal recessiveAntenatal, Neonatal

Ассоциированные гены (1)

GPX4
glutathione peroxidase 4
Disease-causing germline mutation(s) in
OMIM: 138322

Фенотипы (25)

Очень частый (80–99%)15
HP:0000772Abnormal rib morphology
HP:0000782Abnormality of the scapula
HP:0000926Platyspondyly
HP:0001678Atrioventricular block
HP:0002657Spondylometaphyseal dysplasia
HP:0002750Delayed skeletal maturation
HP:0003085Long fibula
HP:0003498Disproportionate short stature
HP:0004279Short palm
HP:0004991Rhizomelic arm shortening
HP:0005871Metaphyseal chondrodysplasia
HP:0006543Cardiorespiratory arrest
HP:0008786Iliac crest serration
HP:0010049Short metacarpal
HP:0011675Arrhythmia
Частый (30–79%)5
HP:0000262Turricephaly
HP:0000774Narrow chest
HP:0001290Generalized hypotonia
HP:0001321Cerebellar hypoplasia
HP:0002093Respiratory insufficiency
Периодический (5–29%)5
HP:0001274Agenesis of corpus callosum
HP:0001302Pachygyria
HP:0005616Accelerated skeletal maturation
HP:0010579Cone-shaped epiphysis
HP:0012819Myocarditis

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