Neurohormonal modulation for treatment of cardiac involvement in dystrophinopathies and mitochondrial disease

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Abstract

Mutations in either the nuclear or the mitochondrial genome can lead to structural and functional changes within the skeletal muscles. These genetic skeletal myopathies are rare, although not infrequent when their cumulative incidence is considered. Dystrophinopathies (Duchenne and Becker muscular dystrophies) and mitochondrial disease are some of the most frequent clinical entities, and those developing heart failure more frequently. Neurohormonal antagonism represents the cornerstone of heart failure management, even though its role in the prevention and treatment of heart failure in patients with dystrophinopathies or mitochondrial disorders remains undefined. In the present paper we will summarise current available evidence on this topic. Particular attention will be devoted to Duchenne muscular dystrophy, and to the approaches modulating neurohormonal function by targeting the skeletal muscle.

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