COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING
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United States of America Patent
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Issued Date -
Mar 30, 2017
app pub date -
Sep 16, 2016
filing date -
Jun 23, 2005
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Abstract
Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
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