TargetMol

Branaplam mesylate (1562338-42-4 free base)

Product Code:
 
TAR-T26893
Product Group:
 
Inhibitors and Activators
Supplier:
 
TargetMol
Regulatory Status:
 
RUO
Shipping:
 
cool pack
Storage:
 
-20°C
1 / 1
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TAR-T26893-500mg500mgEnquire
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Further Information

Bioactivity:
Branaplam is a highly potent, selective and orally active SMN2 splicing modulator. Branaplam increases the amount of functional SMN protein produced by the SMN2 gene through modifying its splicing.
CAS:
T26893
Molecular Weight:
489.59
Purity:
0.98
SMILES:
O=C1C=C(C2=CNN=C2)C=C/C1=C3C=CC(OC4CC(C)(C)NC(C)(C)C4)=NN\3.OS(=O)(C)=O

References

Cheung AK, Hurley B, Kerrigan R, Shu L, Chin DN, Shen Y, O'Brien G, Sung MJ, Hou Y, Axford J, Cody E, Sun R, Fazal A, Tomlinson RC, Jain M, Deng L, Hoffmaster K, Song C, Van Hoosear M, Shin Y, Servais R, Towler CS, Hild M, Curtis D, Dietrich WF, Hamann LG, Briner K, Chen KS, Kobayashi D, Sivasankaran R, Dales NA. Discovery of small molecule splicing modulators of survival motor neuron-2 (SMN2) for the treatment of Spinal Muscular Atrophy (SMA). J Med Chem. 2018 Nov 8. doi: 10.1021/acs.jmedchem.8b01291. [Epub ahead of print] PubMed PMID: 30407821. Stam M, Wadman RI, Wijngaarde CA, Bartels B, Asselman FL, Otto LAM, Goedee HS, Habets LE, de Groot JF, Schoenmakers MAGC, Cuppen I, van den Berg LH, van der Pol WL. Protocol for a phase II, monocentre, double-blind, placebo-controlled, cross-over trial to assess efficacy of pyridostigmine in patients with spinal muscular atrophy types 2-4 (SPACE trial). BMJ Open. 2018 Jul 30;8(7):e019932. doi: 10.1136/bmjopen-2017-019932. PubMed PMID: 30061431; PubMed Central PMCID: PMC6067401. Calder AN, Androphy EJ, Hodgetts KJ. Small Molecules in Development for the Treatment of Spinal Muscular Atrophy. J Med Chem. 2016 Nov 23;59(22):10067-10083. Epub 2016 Aug 16. Review. PubMed PMID: 27490705; PubMed Central PMCID: PMC5744254.