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ATTRV122I amyloidosis

ORPHA:85451Clinical subtypeAutosomal dominantAdult

Ассоциированные гены (1)

TTR
transthyretin
Disease-causing germline mutation(s) in
OMIM: 176300

Фенотипы (25)

Очень частый (80–99%)5
HP:0001692Atrial arrhythmia
HP:0003115Abnormal EKG
HP:0030843Cardiac amyloidosis
HP:0031185Increased circulating NT-proBNP concentration
HP:0410174Increased troponin T level in blood
Частый (30–79%)6
HP:0001639Hypertrophic cardiomyopathy
HP:0001640Cardiomegaly
HP:0001723Restrictive cardiomyopathy
HP:0005150Abnormal atrioventricular conduction
HP:0011675Arrhythmia
HP:0012664Reduced left ventricular ejection fraction
Периодический (5–29%)11
HP:0001297Stroke
HP:0001635Congestive heart failure
HP:0001681Angina pectoris
HP:0001712Left ventricular hypertrophy
HP:0001903Anemia
HP:0001907Thromboembolism
HP:0003416Spinal canal stenosis
HP:0012185Constrictive median neuropathy
HP:0012332Abnormal autonomic nervous system physiology
HP:0025028Abnormality of enteric nervous system morphology
HP:0100550Tendon rupture
Очень редкий (1–4%)3
HP:0001271Polyneuropathy
HP:0001650Aortic valve stenosis
HP:0009830Peripheral neuropathy

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