NVS-SM2 is a potent, orally bioavailable, small-molecule enhancer of SMN2 splicing. NVS-SM2 binds to and stabilizing the 5′ss RNA complex. NVS-SM2 shows increased exon 7 inclusion and upregulated SMN protein expression in both SMA patient fibroblasts and SMNΔ7 5025-mouse myoblasts. References: Calder AN, Androphy EJ, Hodgetts KJ. Small Molecules in Development for the Treatment of Spinal Muscular Atrophy. J Med Chem. 2016 Aug 16. [Epub ahead of print] PubMed PMID: 27490705.

纯度：≥98%

CAS：1562333-92-9