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(Circulation. 2005;112:2756-2758.)
© 2005 American Heart Association, Inc.

Editorial

Evolving Therapeutic Strategies for Dystrophinopathies

Potential for Conflict Between Cardiac and Skeletal Needs

Steven D. Colan, MD

From the Department of Cardiology, Children’s Hospital Boston, Department of Pediatrics, Harvard Medical School, Boston, Mass.

Correspondence to Steven D. Colan, MD, Department of Cardiology, Children’s Hospital Boston, Department of Pediatrics, Harvard Medical School, 300 Longwood Ave, Boston, MA 02115.

Key Words: Editorials • cardiomyopathy • myocardium • muscular dystrophy

An extract of the first 250 words of the full text is provided, because this article has no abstract.

The importance of cardiomyopathy to the clinical course of the dystrophinopathies varies according to the nature of the dystrophin defect. In Becker muscular dystrophy (BMD), cardiac involvement is often the most important determinant of clinical status and long-term outcome. For the Duchenne muscular dystrophy (DMD) patient, cardiac manifestations are often masked by inactivity and respiratory muscle compromise. However, the increasing use of ventilatory assist devices has allowed cardiac failure to emerge as a more prevalent feature of the disease.¹ Furthermore, a wide range of therapeutic interventions to improve skeletal muscle strength are being explored,² some of which may not have equivalent benefit for cardiac muscle. The article by Jefferies et al³ is therefore timely, reporting the potential cardiac benefits of early treatment of patients with DMD and BMD with afterload-reducing therapy. These authors present a retrospective review of the outcome of their patients with DMD or BMD who were managed according to a clinical treatment protocol that included initiation of ACE inhibitor (ACEI) therapy at the time of first recognition of ventricular dysfunction. ß-Blocker (BB) therapy was added for patients who had no evidence of improvement in their ventricular function after 3 months. There are at least 3 important findings in their reported experience. First, there were no significant adverse clinical effects attributable to medical therapy. Although these agents have a remarkable safety profile overall, neuromuscular diseases in general are notorious for unanticipated pharmacological responses, and this result is therefore reassuring. Second, over the average follow-up duration of 3.3 years, . . . [Full Text of this Article]