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Small Molecules acting on Myofilaments as Treatments for Heart and Skeletal Muscle Diseases.


ABSTRACT: Hypertrophic cardiomyopathy (HCM) and dilated cardiomyopathy (DCM) are the most prevalent forms of the chronic and progressive pathological condition known as cardiomyopathy. These diseases have different aetiologies; however, they share the feature of haemodynamic abnormalities, which is mainly due to dysfunction in the contractile proteins that make up the contractile unit known as the sarcomere. To date, pharmacological treatment options are not disease-specific and rather focus on managing the symptoms, without addressing the disease mechanism. Earliest attempts at improving cardiac contractility by modulating the sarcomere indirectly (inotropes) resulted in unwanted effects. In contrast, targeting the sarcomere directly, aided by high-throughput screening systems, could identify small

SUBMITTER: Alsulami K 

PROVIDER: S-EPMC7767104 | biostudies-literature | 2020 Dec

REPOSITORIES: biostudies-literature