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Pseudopseudohypoparathyroidism

ORPHA:79445DiseaseAutosomal dominantAll ages

Ассоциированные гены (1)

GNAS
GNAS complex locus
Disease-causing germline mutation(s) (loss of function) in
OMIM: 139320

Фенотипы (18)

Частый (30–79%)8
HP:0000311Round face
HP:0001156Brachydactyly
HP:0004322Short stature
HP:0009237Short 5th finger
HP:0010044Short 4th metacarpal
HP:0010047Short 5th metacarpal
HP:0010743Short metatarsal
HP:0011986Ectopic ossification
Периодический (5–29%)7
HP:0000750Delayed speech and language development
HP:0001249Intellectual disability
HP:0001511Intrauterine growth retardation
HP:0001513Obesity
HP:0004180Short distal phalanx of the 3rd finger
HP:0009650Short distal phalanx of the thumb
HP:0025027Osteoma cutis
Исключён (0%)3
HP:0002901Hypocalcemia
HP:0002905Hyperphosphatemia
HP:0003165Elevated circulating parathyroid hormone level

Эпидемиология (1)

Point prevalence
Unknown
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы