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Albers-Schönberg osteopetrosis

ORPHA:53Malformation syndromeAutosomal dominantChildhood

Ассоциированные гены (1)

CLCN7
chloride voltage-gated channel 7
Disease-causing germline mutation(s) in
OMIM: 602727

Фенотипы (36)

Очень частый (80–99%)17
HP:0000256Macrocephaly
HP:0000944Abnormal metaphysis morphology
HP:0001369Arthritis
HP:0001373Joint dislocation
HP:0002007Frontal bossing
HP:0002653Bone pain
HP:0002754Osteomyelitis
HP:0002757Recurrent fractures
HP:0002758Osteoarthritis
HP:0005789Generalized osteosclerosis
HP:0005916Abnormal metacarpal morphology
HP:0005930Abnormality of epiphysis morphology
HP:0006824Cranial nerve paralysis
HP:0007626Mandibular osteomyelitis
HP:0009882Short distal phalanx of finger
HP:0010628Facial palsy
HP:0010885Avascular necrosis
Частый (30–79%)7
HP:0000164Abnormality of the dentition
HP:0000648Optic atrophy
HP:0001903Anemia
HP:0002650Scoliosis
HP:0002857Genu valgum
HP:0004322Short stature
HP:0012378Fatigue
Периодический (5–29%)10
HP:0000238Hydrocephalus
HP:0000365Hearing impairment
HP:0000618Blindness
HP:0000670Carious teeth
HP:0001873Thrombocytopenia
HP:0001881Abnormal leukocyte morphology
HP:0002901Hypocalcemia
HP:0005746Osteosclerosis of the base of the skull
HP:0012145Abnormality of multiple cell lineages in the bone marrow
HP:0030757Tooth abscess
Очень редкий (1–4%)2
HP:0000505Visual impairment
HP:0001293Cranial nerve compression

Эпидемиология (4)

Point prevalence
1-9 / 100 000
Worldwide
Point prevalence
1-9 / 100 000
Denmark
Point prevalence
1-9 / 1 000 000
Brazil
Point prevalence
1-9 / 100 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы