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Carney-Stratakis syndrome

ORPHA:97286DiseaseAutosomal dominantAdolescent, Adult

Ассоциированные гены (4)

SDHA
succinate dehydrogenase complex flavoprotein subunit A
Disease-causing germline mutation(s) in
OMIM: 600857
SDHB
succinate dehydrogenase complex iron sulfur subunit B
Disease-causing germline mutation(s) (loss of function) in
OMIM: 185470
SDHC
succinate dehydrogenase complex subunit C
Disease-causing germline mutation(s) (loss of function) in
OMIM: 602413
SDHD
succinate dehydrogenase complex subunit D
Disease-causing germline mutation(s) (loss of function) in
OMIM: 602690

Фенотипы (10)

Очень частый (80–99%)2
HP:0002668Paraganglioma
HP:0100723Gastrointestinal stroma tumor
Частый (30–79%)8
HP:0000360Tinnitus
HP:0000365Hearing impairment
HP:0001824Weight loss
HP:0002015Dysphagia
HP:0002027Abdominal pain
HP:0002239Gastrointestinal hemorrhage
HP:0005214Intestinal obstruction
HP:0006824Cranial nerve paralysis

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