Efficacy of sirolimus in treating tuberous sclerosis and lymphangioleiomyomatosis

E Paul, E Thiele - New England Journal of Medicine, 2008 - Mass Medical Soc
E Paul, E Thiele
New England Journal of Medicine, 2008Mass Medical Soc
Owing to their immunosuppressive and antiproliferative effects, sirolimus (also called
rapamycin) and related drugs are being evaluated as part of many transplant
immunosuppresion regimens, as well as for a plethora of medical conditions such as type 1
diabetes, macular degeneration, coronary artery disease, and metastatic or refractory
cancers of the breast, prostate, lung, and liver, to name but a few. The effects of sirolimus are
mediated by its inhibition of the curiously named cytoplasmic protein mammalian target of …
Owing to their immunosuppressive and antiproliferative effects, sirolimus (also called rapamycin) and related drugs are being evaluated as part of many transplant immunosuppresion regimens, as well as for a plethora of medical conditions such as type 1 diabetes, macular degeneration, coronary artery disease, and metastatic or refractory cancers of the breast, prostate, lung, and liver, to name but a few.
The effects of sirolimus are mediated by its inhibition of the curiously named cytoplasmic protein mammalian target of rapamycin (mTOR), a ubiquitous serine–threonine kinase that is intimately involved in the regulation of protein synthesis, cell growth, cytoskeletal organization, and other . . .
The New England Journal Of Medicine