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Updated: Sep 15, 2025

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
Published on: May 11, 2018
Novel Heterocyclic Compounds as SMN2 Modulators for Treating Spinal Muscular Atrophy Via Modulation of SMN2 Splicing
1Smith, Gambrell & Russell LLP, 1105 W. Peachtree Street NE, Atlanta, Georgia 30309, United States.
Abstract:
Provided herein are novel heterocyclic compounds as SMN2 modulators, pharmaceutical compositions, use of such compounds in treating spinal muscular atrophy (SMA) via modulation of SMN2 splicing, and processes for preparing such compounds.
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