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Therapeutic Strategies for Treatment of Pulmonary Lymphangioleiomyomatosis (LAM).

Vera P Krymskaya
Published in: Expert opinion on orphan drugs (2014)
Targeting mTOR activation with rapamycin analogs sirolimus and everolimus are awaiting approval by the FDA for treatment of LAM. A number of other treatment options have been investigated and are currently tested in clinical trials to target LAM cell survival and metastasis. Key remaining and poorly understood areas for development and validation of therapeutic targeting in LAM are destruction of lungs, pathological lymphangiogenesis, and hormonal regulation. Future will reveal whether they could be targeted therapeutically.
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