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.

Real-world evidence study on the long-term safety of everolimus in patients with TSC

The TuberOus SClerosis registry to increase disease Awareness (TOSCA) Post-Authorization Safety Study (PASS) was a non-interventional, multicenter, safety study that assessed the long-term safety of everolimus in patients with TSC in the European Union.

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Antiepileptic effect and safety profile of Rapamycin in paediatric patients with TSC

Treatment with mTOR pathway inhibitors is important in drug resistant epilepsy (DRE). This study sought to evaluate the safety and efficacy of 12 months of Rapamycin treatment in 32 paediatric patients aged from 11 months to 14 years, all of whom had drug-resistant TSC-associated epilepsy.

DISCLAIMER

This information is intended to provide some insights into recent TSC-related research. 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.

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