mTOR Inhibitor Treatments in TSC
The term mTOR inhibitor refers to a group of TSC-specific medicines.
As with all information published on this site, this page is intended to provide basic information about TSC (Tuberous Sclerosis Complex). It is not intended to, and it should not, constitute medical or other advice. Readers are warned not to take any action without first seeking medical advice.
Signs, Symptoms and
Last updated: 21 February 2023
Reviewed by: Dr Sean Kennedy, Paediatric Nephrologist, Sydney Children’s Hospital; Dr Zebunessa Rahman, Consultant Neurologist, Westmead Hospital; Dr Matthew Sypek, Consultant Nephrologist, Royal Childrens Hospital and Royal Melbourne Hospital; A/Prof Orli Wargon, Paediatric Dermatologist, Sydney Children’s Hospital.
- European Medicines Agency. EPAR Summary for the Public: Votubia (Everolmius). Viewed 1st August, 2013. https://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/002311/human_med_001484.jsp
- Franz, D.N., et al., Efficacy and safety of everolimus for subependymal giant cell astrocytomas associated with tuberous sclerosis complex (EXIST-1): a multicentre, randomised, placebo-controlled phase 3 trial. Lancet, 2013. 381(9861): p. 125-32.
- Bissler, J.J., et al., Everolimus for angiomyolipoma associated with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis (EXIST-2): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet, 2013. 381(9869): p. 817-24.
- McCormack, F.X., et al., Efficacy and safety of sirolimus in lymphangioleiomyomatosis. The New England Journal of Medicine, 2011. 364: p. 1595-1606.