| Literature DB >> 33638459 |
Cecilie Johannessen Landmark1,2,3, Heidrun Potschka4, Stéphane Auvin5,6, Jo M Wilmshurst7, Svein I Johannessen2,3, Dorothée Kasteleijn-Nolst Trenité8, Elaine C Wirrell9.
Abstract
Developmental and epileptic encephalopathies (DEEs) are among the most challenging of all epilepsies to manage, given the exceedingly frequent and often severe seizure types, pharmacoresistance to conventional antiseizure medications, and numerous comorbidities. During the past decade, efforts have focused on development of new treatment options for DEEs, with several recently approved in the United States or Europe, including cannabidiol as an orphan drug in Dravet and Lennox-Gastaut syndromes and everolimus as a possible antiepileptogenic and precision drug for tuberous sclerosis complex, with its impact on the mammalian target of rapamycin pathway. Furthermore, fenfluramine, an old drug, was repurposed as a novel therapy in the treatment of Dravet syndrome. The evolution of new insights into pathophysiological processes of various DEEs provides possibilities to investigate novel and repurposed drugs and to place them into the context of their role in future management of these patients. The purpose of this review is to provide an overview of these new medical treatment options for the DEEs and to discuss the clinical implications of these results for improved treatment.Entities:
Keywords: Dravet syndrome; Lennox-Gastaut syndrome; antiseizure medications; cannabidiol; drug repurposing; everolimus; fenfluramine; orphan drugs; tuberous sclerosis
Year: 2021 PMID: 33638459 DOI: 10.1111/epi.16849
Source DB: PubMed Journal: Epilepsia ISSN: 0013-9580 Impact factor: 5.864