Abstract

Patients with Duchenne muscular dystrophy (DMD) lack expression of the protein dystrophin due to mutations in the X-linked dystrophin gene. Patients suffer from severe skeletal muscle pathologies as well as cardiomyopathy, congestive heart failure, and arrhythmias. Like humans, dystrophin-deficient mice (mdx mice) show cardiac dysfunction. Adamo et al (Proc Natl Acad Sci U S A 2010;107:19079 PMID 20956307) showed that sildenafil citrate (Viagra), a phosphodiesterase 5 (PDE5) inhibitor, treatment reduces functional deficits in the cardiac performance of aged mdx mice, with no effect on normal cardiac function in wild-type (WT) controls.

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