Study shows targeted therapy importance
Congenital heart diseases affect approximately one in 100 patients, making them the most common type of birth defect and the number-one cause of pediatric deaths.
Now a new study shows that the mTOR inhibitor drug rapamycin can reverse cardiac muscle damage in a mouse model of the congenital disease LEOPARD syndrome. The study not only identifies the first possible medical treatment for this rare condition, but demonstrates the importance of targeted therapies in managing congenital diseases.
The research, led by BIDMC investigators, is published in the March issue of the Journal of Clinical Investigation (JCI.)
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