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Schneckenbecken dysplasia

ORPHA:3144Malformation syndromeAutosomal recessiveAntenatal

Ассоциированные гены (2)

SLC35D1
solute carrier family 35 member D1
Disease-causing germline mutation(s) (loss of function) in
OMIM: 610804
INPPL1
inositol polyphosphate phosphatase like 1
Disease-causing germline mutation(s) in
OMIM: 600829

Фенотипы (26)

Очень частый (80–99%)17
HP:0000256Macrocephaly
HP:0000272Malar flattening
HP:0000470Short neck
HP:0000773Short ribs
HP:0000774Narrow chest
HP:0000882Hypoplastic scapulae
HP:0000895Lateral clavicle hook
HP:0000944Abnormal metaphysis morphology
HP:0000946Hypoplastic ilia
HP:0001004Lymphedema
HP:0001561Polyhydramnios
HP:0002983Micromelia
HP:0003038Fibular hypoplasia
HP:0003312Abnormal form of the vertebral bodies
HP:0008479Hypoplastic vertebral bodies
HP:0008873Disproportionate short-limb short stature
HP:0012107Increased fibular diameter
Частый (30–79%)4
HP:0000028Cryptorchidism
HP:0000268Dolichocephaly
HP:0001231Abnormal fingernail morphology
HP:0001800Hypoplastic toenails
Периодический (5–29%)5
HP:0000175Cleft palate
HP:0000947Dumbbell-shaped long bone
HP:0005019Diaphyseal thickening
HP:0005616Accelerated skeletal maturation
HP:0008108Advanced tarsal ossification

Эпидемиология (1)

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