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BRESEK syndrome

ORPHA:85284Malformation syndromeX-linked dominantChildhood

Ассоциированные гены (1)

MBTPS2
membrane bound transcription factor peptidase, site 2
Disease-causing germline mutation(s) in
OMIM: 300294

Фенотипы (29)

Очень частый (80–99%)13
HP:0000028Cryptorchidism
HP:0000089Renal hypoplasia
HP:0000238Hydrocephalus
HP:0000252Microcephaly
HP:0000369Low-set ears
HP:0000411Protruding ear
HP:0000609Optic nerve hypoplasia
HP:0001263Global developmental delay
HP:0001510Growth delay
HP:0001596Alopecia
HP:0005343Hypoplasia of the bladder
HP:0010864Intellectual disability, severe
HP:0012443Abnormality of brain morphology
Частый (30–79%)15
HP:0000076Vesicoureteral reflux
HP:0000110Renal dysplasia
HP:0000175Cleft palate
HP:0000365Hearing impairment
HP:0000444Convex nasal ridge
HP:0000568Microphthalmia
HP:0000612Iris coloboma
HP:0001162Postaxial hand polydactyly
HP:0001357Plagiocephaly
HP:0001511Intrauterine growth retardation
HP:0002251Aganglionic megacolon
HP:0002650Scoliosis
HP:0002937Hemivertebrae
HP:0003811Neonatal death
HP:0008734Decreased testicular size
Периодический (5–29%)1
HP:0008064Ichthyosis

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