OPKO Health, Inc. (NYSE Amex: OPK) announced today that it has been awarded a grant from the Dravet Syndrome Foundation to support the Company's efforts to develop potential therapeutic agents to treat a rare genetic disorder, Dravet Syndrome, also called severe myoclonic epilepsy of infancy, a severe form of epilepsy that usually appears during the first year of life.
OPKO is using its broad proprietary platform technology that utilizes specifically designed oligonucleotides to up-regulate a targeted gene through inhibition of Natural Antisense Transcripts (NATs) to increase the production of endogenous SCN1A (also referred to as sodium channel) protein. More than 75% of children who have been diagnosed with Dravet syndrome have a defective SCN1A gene and, as a result, the defective gene does not produce enough functional protein, leading to a diseased state.
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