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F1000 Med Rep. 2009 Dec 9;1. pii: 93. doi: 10.3410/M1-93.

Therapeutic options for lymphangioleiomyomatosis (LAM): where we are and where we are going.

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Translational Medicine Branch Building 10, Room 6D05, MSC 1590 National Heart, Lung, and Blood Institute, National Institutes of Health Bethesda, MD 20892-1590 USA.


Lymphangioleiomyomatosis (LAM), a multisystem disease affecting predominantly premenopausal and middle-aged women, causes progressive respiratory failure due to cystic lung destruction and is associated with lymphatic and kidney tumors. In the past, the treatment of LAM comprised exclusively anti-estrogen and related hormonal therapies. These treatments, however, have not been proven effective. In this article, we discuss new findings regarding the molecular mechanisms involved in the regulation of LAM cell growth, which may offer opportunities to develop effective and targeted therapeutic agents.

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