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Effectiveness and safety of everolimus treatment in patients with tuberous sclerosis complex in real-world clinical practice

2 Dec 2023

Abstract excerpt

BACKGROUND: The randomised double-blinded placebo-controlled EXIST-1-3 studies have showed everolimus effective with adverse effects reported as acceptable in treatment of symptoms in patients with tuberous sclerosis complex (TSC), although evidence of outcomes in clinical practice remains limited. This study aimed to investigate, in clinical practice, the effectiveness and safety of everolimus for epilepsy,...

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PMID: 38042867
DOI: 10.1186/s13023-023-02982-1

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