Expanding the therapeutic role of highly purified cannabidiol in monogenic epilepsies: A multicenter real-world study.

ABSTRACT:

Objective

This real-world, retrospective, multicenter study aims to investigate the effectiveness of highly purified cannabidiol (CBD) in a large cohort of patients with epilepsy of genetic etiology due to an identified monogenic cause. Additionally, we examine the potential relationship between specific genetic subgroups and treatment response.

Methods

This study was conducted across 27 epilepsy centers and included patients with monogenic epileptic disorders (pathogenic or likely pathogenic variants) who were treated with highly purified CBD for at least 3 months.

Results

A total of 266 patients (135 females, 50.8%) with monogenic epilepsies were included with a median age at CBD initiation of 12 years (interquartile range [IQR] = 7-19) and a median follow-up durati

SUBMITTER: Cerulli Irelli E 

PROVIDER: S-EPMC12291005 | biostudies-literature | 2025 Jul

REPOSITORIES: biostudies-literature