Ionis Pharmaceuticals, Inc.
COMPOUNDS AND METHODS FOR MODULATION OF SMN2
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Abstract:
Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of spinal muscular atrophy.
Status:
Application
Type:
Utility
Filling date:
18 Dec 2020
Issue date:
10 Mar 2022