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Drug Approved for LAM Illustrates How Patient Organizations Can Partner With Researchers


 

References

On May 28, FDA approved Rapamune (sirolimus) as a treatment for LAM (lymphangioleiomyomatosis), a rare, progressive and potentially fatal lung disease that affects primarily women of childbearing age. It is the first treatment to be approved for LAM. The LAM Foundation, a patient advocacy organization established by the parents of a young woman who died of LAM, worked closely with researchers at the University of Cincinnati Medical School on research that ultimately led to development of this treatment by Wyeth Pharmaceuticals, Inc. An editorial in the New England Journal of Medicine a few years ago cited the partnership between the patient organization and researchers as a model for advancing treatments for rare diseases. Read the FDA press release about the drug approval.

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