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Hypocalcemic vitamin D-resistant rickets

ORPHA:93160DiseaseAutosomal recessiveInfancy, Neonatal

Ассоциированные гены (1)

VDR
vitamin D receptor
Disease-causing germline mutation(s) in
OMIM: 601769

Фенотипы (28)

Очень частый (80–99%)11
HP:0000843Hyperparathyroidism
HP:0001373Joint dislocation
HP:0002148Hypophosphatemia
HP:0002653Bone pain
HP:0002749Osteomalacia
HP:0002757Recurrent fractures
HP:0002797Osteolysis
HP:0002901Hypocalcemia
HP:0003330Abnormal bone structure
HP:0012062Bone cyst
HP:0100670Rough bone trabeculation
Частый (30–79%)13
HP:0000268Dolichocephaly
HP:0000765Abnormal thorax morphology
HP:0000787Nephrolithiasis
HP:0000944Abnormal metaphysis morphology
HP:0000951Abnormality of the skin
HP:0001288Gait disturbance
HP:0001596Alopecia
HP:0002970Genu varum
HP:0003272Abnormality of the hip bone
HP:0003312Abnormal form of the vertebral bodies
HP:0004322Short stature
HP:0006323Premature loss of primary teeth
HP:0009124Abnormal adipose tissue morphology
Периодический (5–29%)4
HP:0000164Abnormality of the dentition
HP:0002007Frontal bossing
HP:0002650Scoliosis
HP:0002857Genu valgum

Эпидемиология (1)

Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы