Branaplam

  • Chemical Properties
CAS No. 1562338-42-4 Cat. No. BCP19909
Name Branaplam
Synonyms LMI-070;LMI070;LMI 070;NVS-SM1;
Formula C22H27N5O2 M. Wt 393.48
  • Biological Activity
Description Branaplam, also known as NVS-SM1 and LMI-070, is a highly potent, selective and orally active small molecule SMN2 splicing modulator as a potential treatment for SMA Type 1 (Spinal Muscular Atrophy) .NVS-SM1 works by increasing the amount of functional SMN protein produced by the “back-up” gene, SMN2, through modifying its splicing.
Related Products 1562338-39-9(Branaplam hydrochloride)
Pathways Cell Cycle/DNA Damage 
Targets DNA/RNA Synthesis 

Structure

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