Goal 3: Advance Translational Research

To find specific medical therapies to treat the wide array of human vascular malformations and vascular tumors.

Vascular malformations and vascular tumors, together referred to as vascular anomalies, comprise a complex and wide array of diseases in which there is a fundamental disruption in blood and lymphatic vasculature. The lesions disrupt organ function, destroy tissue, cause bleeding, increase infections and can threaten life. At present, there are some medical therapies but none are specifically targeted to an underlying molecular defect. For many there is no medical therapy available at all and there is little to no insight that identifies the cellular culprit or the molecular/biochemical defect. Thus, basic knowledge on which to begin developing targeted therapies is lacking.

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Idea No. 929