Clinical Trial Design for Disease-Modifying Therapies for Genetic Epilepsies.

ABSTRACT: Although trials with anti-seizure medications (ASMs) have not shown clear anti-epileptogenic or disease-modifying activity in humans to date, rapid advancements in genomic technology and emerging gene-mediated and gene replacement options offer hope for the successful development of disease-modifying therapies (DMTs) for genetic epilepsies. In fact, more than 26 potential DMTs are in various stages of preclinical and/or clinical development for genetic syndromes associated with epilepsy. The scope of disease-modification includes but is not limited to effects on the underlying pathophysiology, the condition's natural history, epilepsy severity, developmental achievement, function, behavior, sleep, and quality of life. While conventional regulatory clinical trials for epilepsy therapeutics

SUBMITTER: Brock DC 

PROVIDER: S-EPMC8609073 | biostudies-literature | 2021 Jul

REPOSITORIES: biostudies-literature