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Autosomal recessive hypophosphatemic rickets

ORPHA:289176DiseaseAutosomal recessiveChildhood, Infancy

Ассоциированные гены (2)

DMP1
dentin matrix acidic phosphoprotein 1
Disease-causing germline mutation(s) in
OMIM: 600980
ENPP1
ectonucleotide pyrophosphatase/phosphodiesterase 1
Disease-causing germline mutation(s) in
OMIM: 173335

Фенотипы (37)

Облигатный (100%)1
HP:0004912Hypophosphatemic rickets
Очень частый (80–99%)25
HP:0000117Renal phosphate wasting
HP:0000407Sensorineural hearing impairment
HP:0000684Delayed eruption of teeth
HP:0001510Growth delay
HP:0002652Skeletal dysplasia
HP:0002653Bone pain
HP:0002749Osteomalacia
HP:0002812Coxa vara
HP:0002814Abnormality of the lower limb
HP:0002970Genu varum
HP:0003020Enlargement of the wrists
HP:0003109Hyperphosphaturia
HP:0004322Short stature
HP:0004576Sclerotic vertebral endplates
HP:0005096Distal femoral bowing
HP:0005764Polyarticular arthritis
HP:0006463Rickets of the lower limbs
HP:0008732Renal hypophosphatemia
HP:0010639Elevated alkaline phosphatase of bone origin
HP:0011001Increased bone mineral density
HP:0011036Abnormality of renal excretion
HP:0012052Low serum calcitriol
HP:0100511Abnormality of vitamin D metabolism
HP:0100559Lower limb asymmetry
HP:0100671Abnormal trabecular bone morphology
Частый (30–79%)8
HP:0001363Craniosynostosis
HP:0002024Malabsorption
HP:0002982Tibial bowing
HP:0003416Spinal canal stenosis
HP:0030757Tooth abscess
HP:0100036Pseudo-fractures
HP:0100686Enthesitis
HP:0100781Abnormality of the sacroiliac joint
Исключён (0%)3
HP:0001250Seizure
HP:0001324Muscle weakness
HP:0003472Hypocalcemic tetany

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