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Isis And Collaborators Alleviate Disease In Animal Models Of Myotonic Dystrophy
Date:8/1/2012

CARLSBAD, Calif., Aug. 1, 2012 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that new preclinical data evaluating antisense compounds to treat myotonic dystrophy type 1 (DM1) were published in the journal Nature.  These new findings show that antisense targeting of a toxic RNA, the disease causing component in DM1, resulted in reversal of disease symptoms that were sustained up to one year after treatment in a mouse model of DM1.

"DM1 is a progressive disease that leads to the gradual loss of muscle function.  Although DM1 is estimated to effect approximately 150,000 patients worldwide, there are no treatments available that could delay the onset of symptoms or slow down the progression of this debilitating disease.  We are encouraged by these early results, which build on the groundbreaking work conducted by Dr. Charles Thornton at the University of Rochester Medical Center and others elucidating the mechanism of DM1.  Using our antisense technology, we and our collaborators were able to target the toxic RNA, remove it and restore normal cell function," said Frank Bennett, Ph.D., Senior Vice President, Research at Isis. 

In the published study, Isis and collaborators from the University of Rochester evaluated antisense compounds in a mouse model of DM1.  Isis designed several antisense compounds to predominantly target an RNA with an expanded triplet repeat sequence characteristic of the genetic defect in DM1.  Researchers confirmed that the antisense compounds entered muscle cells and significantly reduced the toxic RNA, leading to reversal of the disease symptoms that were sustained for up to one year after treatment was discontinued. 

"We are very excited about the potential for antisense to treat severe and rare diseases, which often are not easily targeted with other therapeutic
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SOURCE Isis Pharmaceuticals, Inc.
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