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Familial visceral myopathy

ORPHA:2604DiseaseAutosomal dominantAll ages

Ассоциированные гены (1)

ACTG2
actin gamma 2, smooth muscle
Disease-causing germline mutation(s) in
OMIM: 102545

Фенотипы (23)

Очень частый (80–99%)3
HP:0000021Megacystis
HP:0000072Hydroureter
HP:0000076Vesicoureteral reflux
Частый (30–79%)2
HP:0003270Abdominal distention
HP:0010318Aplasia/Hypoplasia of the abdominal wall musculature
Периодический (5–29%)18
HP:0000358Posteriorly rotated ears
HP:0000175Cleft palate
HP:0000252Microcephaly
HP:0000311Round face
HP:0000337Broad forehead
HP:0000347Micrognathia
HP:0000426Prominent nasal bridge
HP:0000463Anteverted nares
HP:0000774Narrow chest
HP:0000843Hyperparathyroidism
HP:0001166Arachnodactyly
HP:0001387Joint stiffness
HP:0001537Umbilical hernia
HP:0001798Anonychia
HP:0002251Aganglionic megacolon
HP:0003363Abdominal situs inversus
HP:0030680Abnormal cardiovascular system morphology
HP:0100490Camptodactyly of finger

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