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Novel therapeutic options for Dravet and Lennox-Gastaut syndrome

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Epileptic encephalopathies affect around one in 2000 children and often receive supportive treatment.Therefore, novel strategies to improve their overall neurological outcome are needed. Dravet syndrome (DS) is a genetic condition… Click to show full abstract

Epileptic encephalopathies affect around one in 2000 children and often receive supportive treatment.Therefore, novel strategies to improve their overall neurological outcome are needed. Dravet syndrome (DS) is a genetic condition that is primarily associated with loss-of-function mutations in SCN1A, encoding for voltage-gated sodium channels and resulting in loss of action firing in gamma-aminobutyric acid (GABA)-ergic interneurons [1]. By contrast, Lennox-Gastaut syndrome (LGS) has a relatively heterogeneous etiology: in 65–75% of patients, the cause is identifiable (genetic, structural, or metabolic) but unknown in others [1].Patients withthese syndromes are typically refractory to treatment except occasional, short remission periods. Data deriving from new studies have suggested the use as an antiepileptic drug of an old drug like fenfluramine (FFA) which was previously used as an appetite suppressant [2]. Moreover, cannabidiol (CBD) has some evidence of efficacy and an adequate safety profile when used as adjunctive therapy in children and young adults with treatment-resistant epilepsies, including LGS and DS [3,4].We herein focuson available data about the use of FFA and CBD, discussing the most solid clinical evidence supporting their use as medications in LGS and DS.

Keywords: therapeutic options; dravet lennox; lennox gastaut; options dravet; gastaut syndrome; novel therapeutic

Journal Title: Expert Review of Neurotherapeutics
Year Published: 2020

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