NVS-SM2 is a potent, orally active and brain-penetrant SMN2 splicing enhancer with an EC50 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[1][2].
Molecular Weight:
406.52
Purity:
99.15
CAS Number:
[1562333-92-9]
Formula:
C23H30N6O
Target:
DNA/RNA Synthesis
Application Notes:
MCE Product type: Reference compound1
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