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Medical News and Perspectives
May 3, 2006

Drug Shows Promise for Marfan Syndrome

JAMA. 2006;295(17):1990. doi:10.1001/jama.295.17.1990

Scientists using genetics to unravel the molecular basis of a genetic disorder that can cause skeletal malformations and heart abnormalities have discovered that a widely used hypertension drug prevents a deadly complication of the disorder in animals.

A team of scientists at Johns Hopkins University has found that treatment with losartan prevented the development of aortic aneurysm and other defects in a mouse model of Marfan syndrome (Habashi JP et al. Science. 2006;312:117-121). Based on these promising findings and losartan's established safety in humans, the investigators plan to launch clinical trials of the drug as a treatment for the disorder in the fall.

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