Experimental Treatments for Dravet Syndrome

Overview

There is currently no cure for Dravet syndrome, and available treatments are mainly aimed at managing symptoms, specifically seizures that are common in Dravet patients. Researchers are continuing to investigate a number of experimental therapies to treat the disease and to more effectively control seizures. Some of these investigational treatments are summarized below.

EPX-100

EPX-100 is a repurposed antihistamine that is thought to be able to suppress seizures through its action on the serotonin signaling pathways, a mechanism that is different from its anti-histaminic properties. Researchers identified it in a screen of thousands of approved medications that they conducted in a zebrafish model of Dravet syndrome. A Phase 2 trial in Dravet patients is currently underway.

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Zorevunersen

Zorevunersen is an investigational therapy being developed by Stoke Therapeutics for the treatment of Dravet syndrome. It’s administered via intrathecal injections, which are injections into the spinal canal.

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