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