Branaplam hydrochloride (LMI070, NVS-SM1) is a selective and orally active SMN2 splicing modulator that acts directly on SMN with an EC50 value of 20 nM. Branaplam hydrochloride also inhibits hERG channels, demonstrating an IC50 of 6.3 µM. In a mouse model of severe spinal muscular atrophy (SMA), Branaplam hydrochloride significantly increased full-length SMN protein levels and markedly improved survival.
Molecular Weight:
429.95
Purity:
98.45%
CAS Number:
[1562338-39-9]
Formula:
C22H28ClN5O2
Target:
Others|||Potassium Channel|||DNA/RNA Synthesis
T62381
T62381
* VAT and and shipping costs not included. Errors and price changes excepted
