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Multicentric carpo-tarsal osteolysis with or without nephropathy

ORPHA:2774Malformation syndromeAutosomal dominantChildhood

Ассоциированные гены (1)

MAFB
MAF bZIP transcription factor B
Disease-causing germline mutation(s) in
OMIM: 608968

Фенотипы (21)

Очень частый (80–99%)14
HP:0000093Proteinuria
HP:0000325Triangular face
HP:0000347Micrognathia
HP:0000520Proptosis
HP:0001225Wrist swelling
HP:0001288Gait disturbance
HP:0001376Limitation of joint mobility
HP:0001495Carpal osteolysis
HP:0001504Metacarpal osteolysis
HP:0002797Osteolysis
HP:0003019Abnormality of the wrist
HP:0003100Slender long bone
HP:0003457EMG abnormality
HP:0004326Cachexia
Частый (30–79%)2
HP:0000112Nephropathy
HP:0100490Camptodactyly of finger
Периодический (5–29%)5
HP:0000431Wide nasal bridge
HP:0000506Telecanthus
HP:0001561Polyhydramnios
HP:0002714Downturned corners of mouth
HP:0005930Abnormality of epiphysis morphology

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