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Lowry-Wood syndrome

ORPHA:1824DiseaseAutosomal recessiveNeonatal

Ассоциированные гены (1)

RNU4ATAC
RNA, U4atac small nuclear
Disease-causing germline mutation(s) in
OMIM: 601428

Фенотипы (21)

Очень частый (80–99%)5
HP:0000252Microcephaly
HP:0002656Epiphyseal dysplasia
HP:0004322Short stature
HP:0005930Abnormality of epiphysis morphology
HP:0010582Irregular epiphyses
Частый (30–79%)5
HP:0000639Nystagmus
HP:0001249Intellectual disability
HP:0002812Coxa vara
HP:0002829Arthralgia
HP:0007703Abnormality of retinal pigmentation
Периодический (5–29%)11
HP:0000483Astigmatism
HP:0000505Visual impairment
HP:0000926Platyspondyly
HP:0001156Brachydactyly
HP:0001387Joint stiffness
HP:0002750Delayed skeletal maturation
HP:0002999Patellar dislocation
HP:0003042Elbow dislocation
HP:0003083Dislocated radial head
HP:0007370Aplasia/Hypoplasia of the corpus callosum
HP:0100643Abnormality of nail color

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