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WhaTS new? It's cleverolimus

Children and adults with tuberous sclerosis (TS) can have a particularly challenging focal epilepsy to treat. A phase III randomised, double-blind, placebo-controlled trial enrolled 366 people aged 2–65 years to either everolimus or placebo. Everolimus has been studied in the context of reducing lesion growth but not yet as an antiepilepsy agent. The primary outcome was 50% (or more) reduction in seizure frequency. Responses were 15% for the placebo group, 28% for those on low-dose everolimus and 40% for those on a high dose. Fourteen per cent of those on the drug had a serious adverse event, as did 3% on placebo. All normal caveats apply about a short duration of follow-up and that everolimus was …

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