A systems medicine strategy to predict the efficacy of drugs for monogenic epilepsies.

ABSTRACT:

Objective

Monogenic epilepsies are rare but often severe. Because of their rarity, they are neglected by traditional drug developers. Hence, many lack effective treatments. Treatments for a disease can be discovered more quickly and economically by computationally predicting drugs that can be repurposed for it. We aimed to create a computational method to predict the efficacy of drugs for monogenic epilepsies, and to use the method to predict drugs for Dravet syndrome, as (1) it is the archetypal monogenic catastrophic epilepsy; (2) few antiseizure medications are efficacious in Dravet syndrome; and (3) predicting the effect of drugs on Dravet syndrome is challenging, because Dravet syndrome is typically caused by an SCN1A mutation, but some antiseizure medications that are efficac

SUBMITTER: Taweel B 

PROVIDER: S-EPMC10092251 | biostudies-literature | 2022 Dec

REPOSITORIES: biostudies-literature