NVS-SM2 is a potent, orally active and brain-penetrant SMN2 splicing enhancer with an EC 50 of 2 nM for SMN. NVS-SM2 enhances U1-pre-mRNA association. NVS-SM2 promotes exon 7 inclusion and restores normal survival motor neuron (SMN) protein expression. NVS-SM2 can be used for spinal muscular atrophy (SMA) research.
Products | Products > Disease Areas > Neurological, Eye or Ear Disease | Products > Signaling Pathways > Cell Cycle/DNA Damage | Products > Research Areas > Neurological Disease | Products > Signaling Pathways > DNA/RNA Synthesis
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