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Moyamoya angiopathy-short stature-facial dysmorphism-hypergonadotropic hypogonadism syndrome

ORPHA:280679DiseaseX-linked recessiveAll ages

Ассоциированные гены (1)

BRCC3
BRCA1/BRCA2-containing complex subunit 3
Role in the phenotype of
OMIM: 300617

Фенотипы (27)

Очень частый (80–99%)1
HP:0011834Moyamoya phenomenon
Частый (30–79%)17
HP:0000027Azoospermia
HP:0000278Retrognathia
HP:0000316Hypertelorism
HP:0000343Long philtrum
HP:0000518Cataract
HP:0000707Abnormality of the nervous system
HP:0000815Hypergonadotropic hypogonadism
HP:0000822Hypertension
HP:0000823Delayed puberty
HP:0001342Cerebral hemorrhage
HP:0001644Dilated cardiomyopathy
HP:0001999Abnormal facial shape
HP:0002140Ischemic stroke
HP:0002216Premature graying of hair
HP:0004302Functional motor deficit
HP:0004322Short stature
HP:0007970Congenital ptosis
Периодический (5–29%)9
HP:0000369Low-set ears
HP:0000445Wide nose
HP:0000454Flared nostrils
HP:0000490Deeply set eye
HP:0000824Decreased response to growth hormone stimulation test
HP:0001263Global developmental delay
HP:0001324Muscle weakness
HP:0001677Coronaryartery atherosclerosis
HP:0008734Decreased testicular size

Эпидемиология (2)

Cases/families
Worldwide
Point prevalence
<1 / 1 000 000
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы