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SLC39A13-related spondylodysplastic Ehlers-Danlos syndrome

ORPHA:157965Clinical subtypeAutosomal recessiveNeonatal

Ассоциированные гены (1)

SLC39A13
solute carrier family 39 member 13
Disease-causing germline mutation(s) in
OMIM: 608735

Фенотипы (20)

Очень частый (80–99%)8
HP:0000520Proptosis
HP:0000592Blue sclerae
HP:0000963Thin skin
HP:0000974Hyperextensible skin
HP:0000978Bruising susceptibility
HP:0001508Failure to thrive
HP:0002652Skeletal dysplasia
HP:0008848Moderately short stature
Частый (30–79%)11
HP:0000494Downslanted palpebral fissures
HP:0000926Platyspondyly
HP:0000938Osteopenia
HP:0000944Abnormal metaphysis morphology
HP:0001182Tapered finger
HP:0003071Flattened epiphysis
HP:0003370Flat capital femoral epiphysis
HP:0003393Thenar muscle atrophy
HP:0006429Broad femoral neck
HP:0010489Absent palmar crease
HP:0100864Short femoral neck
Периодический (5–29%)1
HP:0001371Flexion contracture

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