Approximately 75% – 90% of people with TSC will have epilepsy at some point in their life. Unfortunately, a significant number do not respond to anti-epileptic medications like Vigabatrin. Everolimus is an mTOR inhibitor medicine that has proven effective as an additional treatment for a person with TSC when other medications have not been able to stop seizures.
The TuberOus SClerosis registry to increase disease Awareness (TOSCA) is perhaps the most significant long-term TSC research study undertaken to date. In this issue of Research Round Up we have included a summary of the study's latest real-world evidence findings on the long-term safety of everolimus in patients with TSC.
We have also included a summary of a recent single centre, open-label study which evaluated safety and anti-epileptic efficacy of 12 months of Rapamycin treatment in paediatric patients with drug-resistant TSC- associated epilepsy.