OPKO Health Announces Grant from Dravet Syndrome Foundation

MIAMI--(BUSINESS WIRE)-- 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.

About the Dravet Syndrome Foundation

The Dravet Syndrome Foundation was founded in September 2009 with the mission to aggressively raise research funds for Dravet syndrome and related conditions, while providing support to affected individuals and families. The Dravet Syndrome Foundation is a volunteer-led organization with no paid employees.

About OPKO Health, Inc.

We are a multi-national biopharmaceutical and diagnostics company that seeks to establish industry-leading positions in large and rapidly growing medical markets by leveraging our discovery, development and commercialization expertise and our novel and proprietary technologies. Our current focus is on conditions with major unmet medical needs including neurological disorders, infectious diseases, oncology and ophthalmologic diseases.

Certain of the statements made in this press release are "forward-looking statements," as that term is defined under the Private Securities Litigation Reform Act of 1995 (PSLRA), which statements may be identified by words such as "expects," "plans," "projects," "will," "may," "anticipates," "believes," "should," "could," "intends," "estimates," and other words of similar meaning, including statements regarding the effectiveness of our technology platform and therapeutic modality for up-regulation of a large number of specific and therapeutically relevant proteins, our ability to develop and commercialize therapeutic agents to treat Dravet syndrome and to use our technology to increase SCN1a protein production. Many factors, including those described in our filings with the Securities and Exchange Commission, could cause actual results or developments to differ materially from those projected or implied in these forward-looking statements, including that clinical trials may fail and not be successful or achieve the expected results or effectiveness, and may not generate data that would support the approval or marketing of these or other products, that others may develop products, including other early stage products which are superior to the products we are developing, and that the products, if developed, may not have advantages over other marketed products. In addition, forward-looking statements may also be adversely affected by risks inherent in funding, developing and obtaining regulatory approvals of new, commercially-viable and competitive products and treatments, general market factors, competitive product development, product availability, federal and state regulations and legislation, the regulatory process for new products and indications, manufacturing issues that may arise, patent positions and litigation, among other factors. The forward-looking statements contained in this press release speak only as of the date the statements were made, and we do not undertake any obligation to update forward-looking statements. We intend that all forward-looking statements be subject to the safe-harbor provisions of the PSLRA.


    Source: OPKO Health, Inc.