Compositions and methods for modulation of SMN2 splicing
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.
Baker; Brenda F. (Carlsbad, CA), Krainer; Adrian R. (Huntington Station, NY), Hua; Yimin (Jericho, NY)
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