Novel Pharmacological Therapy for Dravet Syndrome

Project: Research project

Description

Dravet syndrome is a severe childhood epilepsy for which there are no effective treatments. The syndrome is caused by mutations in the SCN1A gene, which codes a sodium channel that is necessary for normal brain function. A mouse model of Dravet syndrome has been developed that exhibits many features of the human condition and can be used to test new therapies. We recently discovered that a novel chemical compound called GS967 prolongs survival of Dravet syndrome mice, possibly by reducing or eliminating seizures. We wish to investigate how GS967 works to prevent premature death in Dravet syndrome mice. Specifically, we will determine if GS967 prevents seizures and then examine how it affects neuronal electrical
activity. Because GS967 has many properties associated with a successful drug, we hope our studies will guide further development of this and related chemical compounds as potential treatments for Dravet syndrome and other childhood epilepsies for which there are no cures.
StatusFinished
Effective start/end date1/1/1512/31/16

Funding

  • Dravet Syndrome Foundation, Inc. (Agmt Signed 1/30/15)

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Myoclonic Epilepsy
Pharmacology
Epilepsy
Seizures
Therapeutics
Premature Mortality
Sodium Channels
Mutation
Survival
6-(4-(trifluoromethoxy)phenyl)-3-(trifluoromethyl)(1,2,4)triazolo(4,3-a)pyridine
Brain
Pharmaceutical Preparations
Genes