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Mitochondrial DNA-related cardiomyopathy and hearing loss

ORPHA:1349Malformation syndromeMitochondrial inheritanceAdult, Childhood

Ассоциированные гены (1)

MT-TK
mitochondrially encoded tRNA-Lys (AAA/G)
Disease-causing germline mutation(s) in
OMIM: 590060

Фенотипы (30)

Очень частый (80–99%)7
HP:0000407Sensorineural hearing impairment
HP:0001251Ataxia
HP:0001288Gait disturbance
HP:0001324Muscle weakness
HP:0001350Slurred speech
HP:0001639Hypertrophic cardiomyopathy
HP:0011342Mild global developmental delay
Частый (30–79%)14
HP:0000590Progressive external ophthalmoplegia
HP:0000597Ophthalmoparesis
HP:0001268Mental deterioration
HP:0001298Encephalopathy
HP:0001635Congestive heart failure
HP:0001644Dilated cardiomyopathy
HP:0002094Dyspnea
HP:0002151Increased circulating lactate concentration
HP:0003200Ragged-red muscle fibers
HP:0003457EMG abnormality
HP:0003542Increased serum pyruvate
HP:0003546Exercise intolerance
HP:0012514Lower limb pain
HP:0030680Abnormal cardiovascular system morphology
Периодический (5–29%)9
HP:0000822Hypertension
HP:0001012Multiple lipomas
HP:0001347Hyperreflexia
HP:0002373Febrile seizure (within the age range of 3 months to 6 years)
HP:0003326Myalgia
HP:0009126Increased adipose tissue
HP:0009830Peripheral neuropathy
HP:0012378Fatigue
HP:0100749Chest pain

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