Abstract
Objective
Epilepsy is highly prevalent in patients with tuberous sclerosis complex (TSC). Everolimus showed higher efficacy than placebo for seizures in the primary analysis of the EXIST-3 study. Here, we present the long-term outcomes of everolimus at the end of the postextension phase (PEP; data cutoff date: October 25, 2017).
Methods
After completion of the extension phase, patients were invited to continue everolimus in the PEP with everolimus (targeted trough concentration = 5–15 ng/ml, investigator-judged). Efficacy assessments included changes in seizure status during the ...
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