Marfan syndrome pediatric research and clinical trials

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Contact the Heart Center

Boston Children's Hospital is constantly looking for ways to prevent and treat diseases and disorders, including Marfan syndrome.

Problem: Marfan syndrome affects one in 5,000 Americans and weakens the aorta (the blood vessel that carries blood away from the heart to the body), often causing the vessel wall to bulge, forming an aneurysm.

Innovative solution: Traditional treatment for Marfan syndrome often includes beta blockers to lower blood pressure and reduce stress on the aorta. But researchers at Children’s may have found a better solution: We’re testing the blood pressure drug losartan, which may strengthen the aorta and help prevent aortic aneurysms.

Losartan inhibits signaling by transforming growth factor-beta, a protein now known to be overactive in Marfan syndrome, and blocks a cascade of cellular events that weaken aortic tissue. In a landmark 2006 study, losartan completely prevented aortic aneurysms in mice with Marfan syndrome—and even reversed existing aortic damage.

A Phase III trial, one of the largest Marfan studies ever done and co-led by Ronald Lacro, MD, director of Boston Children’s Cardiovascular Genetics Program, has enrolled about 600 patients from 6 months to 25 years of age, as patients in this age group tend to experience the greatest incidence of cardiovascular-related complications.The patients have been randomly assigned to receive either losartan or the beta-blocker atenolol, and are being closely followed for three years.

We are grateful to have been ranked #1 on U.S. News & World Report's list of the best children's hospitals in the nation for the third year in a row, an honor we could not have achieved without the patients and families who inspire us to do our very best for them. Thanks to you, Boston Children's is a place where we can write the greatest children's stories ever told.”
- Sandra L. Fenwick, President and CEO

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