← Назад

Geroderma osteodysplastica

ORPHA:2078Malformation syndromeAutosomal recessiveNeonatal

Ассоциированные гены (2)

GORAB
golgin, RAB6 interacting
Disease-causing germline mutation(s) in
OMIM: 607983
PYCR1
pyrroline-5-carboxylate reductase 1
Disease-causing germline mutation(s) in
OMIM: 179035

Фенотипы (30)

Очень частый (80–99%)12
HP:0001382Joint hypermobility
HP:0000939Osteoporosis
HP:0000963Thin skin
HP:0000974Hyperextensible skin
HP:0001582Redundant skin
HP:0002757Recurrent fractures
HP:0002953Vertebral compression fracture
HP:0003312Abnormal form of the vertebral bodies
HP:0003510Severe short stature
HP:0004568Beaking of vertebral bodies
HP:0004586Biconcave vertebral bodies
HP:0011849Abnormal bone ossification
Частый (30–79%)4
HP:0001252Hypotonia
HP:0001510Growth delay
HP:0002650Scoliosis
HP:0002827Hip dislocation
Периодический (5–29%)14
HP:0000272Malar flattening
HP:0000303Mandibular prognathia
HP:0000478Abnormality of the eye
HP:0000482Microcornea
HP:0000504Abnormality of vision
HP:0000768Pectus carinatum
HP:0000926Platyspondyly
HP:0001256Intellectual disability, mild
HP:0001263Global developmental delay
HP:0001763Pes planus
HP:0001883Talipes
HP:0005930Abnormality of epiphysis morphology
HP:0007495Prematurely aged appearance
HP:0100790Hernia

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