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Lethal Kniest-like dysplasia

ORPHA:2347Malformation syndromeAutosomal recessiveNeonatal

Фенотипы (27)

Частый (30–79%)27
HP:0000175Cleft palate
HP:0000256Macrocephaly
HP:0000260Wide anterior fontanel
HP:0000369Low-set ears
HP:0000470Short neck
HP:0000773Short ribs
HP:0000774Narrow chest
HP:0000907Anterior rib cupping
HP:0000926Platyspondyly
HP:0000946Hypoplastic ilia
HP:0000969Edema
HP:0001156Brachydactyly
HP:0001538Protuberant abdomen
HP:0001561Polyhydramnios
HP:0001623Breech presentation
HP:0001631Atrial septal defect
HP:0001762Talipes equinovarus
HP:0002763Abnormal cartilage morphology
HP:0003015Flared metaphysis
HP:0003174Abnormality of the ischium
HP:0003417Coronal cleft vertebrae
HP:0005026Mesomelic/rhizomelic limb shortening
HP:0005622Broad long bones
HP:0008178Abnormal cartilage matrix
HP:0008479Hypoplastic vertebral bodies
HP:0008890Severe short-limb dwarfism
HP:0012368Flat face

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