title: Treatment of dystrophin cardiomyopathies.
abstract: Treatment of cardiac disease in patients with dystrophinopathies substantially improves outcomes. In this Review, we summarize and discuss findings from the past 20 years and future perspectives for therapeutic options to treat cardiovascular disease in these patients. Their cardiac disease can be subclinical or symptomatic. Presymptomatic treatment with angiotensin-converting-enzyme inhibitors, angiotensin-II-receptor blockers, β-blockers, or mineralocorticoid-receptor antagonists is a well-established method to delay the clinical manifestations of cardiac disease. Treatment of patients with dystrophinopathy and symptomatic cardiac disease, such as heart failure or arrhythmia, follows well-established guidelines for the general treatment of cardiac disease. These treatments improve outcomes, particularly when supported by noncardiovascular measures in the advanced stages of cardiac involvement. Patients with dystrophinopathies and cardiac disease can also benefit from optimal management of scoliosis, noninvasive positive pressure ventilation, and from pain therapy. Molecular therapies for treating cardiac diseases in patients with dystrophinopathies are experimental, but promising.
authors.affiliation: Neurological Department, Krankenanstalt Rudolfstiftung, Postfach 20, 1180 Vienna, Austria.
authors.forename: Josef
authors.initials: J
authors.lastname: Finsterer
authors.affiliation: Department of Pediatrics, Nationwide Children's Hospital, 700 Childrens Drive, Columbus, OH 43205, USA.
authors.forename: Linda
authors.initials: L
authors.lastname: Cripe
mesh_terms: D009202:Cardiomyopathies / Q000188:drug therapy* / Q000209:etiology*; D002317:Cardiovascular Agents / Q000627:therapeutic use*; D016189:Dystrophin / Q000378:metabolism*; D006801:Humans
chemical_list: D002317:Cardiovascular Agents; D016189:Dystrophin