Branaplam: Difference between revisions
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| UNII = P12R69543A |
| UNII = P12R69543A |
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| DrugBank = |
| DrugBank = |
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| ChemSpiderID |
| ChemSpiderID = 34980709 |
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| synonyms = LMI070; NVS-SM1 |
| synonyms = LMI070; NVS-SM1 |
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| StdInChI=1S/C22H27N5O2/c1-21(2)10-16(11-22(3,4)27-21)29-20-8-7-18(25-26-20)17-6-5-14(9-19(17)28)15-12-23-24-13-15/h5-9,12-13,16,25,27H,10-11H2,1-4H3,(H,23,24)/b18-17+ |
| StdInChI=1S/C22H27N5O2/c1-21(2)10-16(11-22(3,4)27-21)29-20-8-7-18(25-26-20)17-6-5-14(9-19(17)28)15-12-23-24-13-15/h5-9,12-13,16,25,27H,10-11H2,1-4H3,(H,23,24)/b18-17+ |
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| StdInChIKey = YIFFDXMJVNKGBL-ISLYRVAYSA-N |
| StdInChIKey = YIFFDXMJVNKGBL-ISLYRVAYSA-N |
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| StdInChI1 = 1S/C22H27N5O2/c1-21(2)10-16(11-22(3,4)27-21)29-20-8-7-18(25-26-20)17-6-5-14(9-19(17)28)15-12-23-24-13-15/h5-9,12-13,16,27-28H,10-11H2,1-4H3,(H,23,24) |
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| StdInChIKey1 = STWTUEAWRAIWJG-UHFFFAOYSA-N |
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Revision as of 10:52, 13 March 2017
Clinical data | |
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Other names | LMI070; NVS-SM1 |
Identifiers | |
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CAS Number | |
PubChem CID | |
ChemSpider | |
UNII | |
Chemical and physical data | |
Formula | C22H27N5O2 |
Molar mass | 393.491 g/mol g·mol−1 |
3D model (JSmol) | |
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Branaplam, also known as LMI070 and NVS-SM1, is a highly potent, selective and orally active small molecule experimental drug being developed by Novartis to treat spinal muscular atrophy (SMA). It works by increasing the amount of functional survival of motor neuron protein produced by the SMN2 gene through modifying its splicing pattern.[1][2]
As of March 2017[update], branaplam is in a phase I–II clinical trial in children with SMA type 1.[3]
References
- ^ Palacino, James; Swalley, Susanne E; Song, Cheng; Cheung, Atwood K; Shu, Lei; Zhang, Xiaolu; Van Hoosear, Mailin; Shin, Youngah; Chin, Donovan N; Keller, Caroline Gubser; Beibel, Martin; Renaud, Nicole A; Smith, Thomas M; Salcius, Michael; Shi, Xiaoying; Hild, Marc; Servais, Rebecca; Jain, Monish; Deng, Lin; Bullock, Caroline; McLellan, Michael; Schuierer, Sven; Murphy, Leo; Blommers, Marcel J J; Blaustein, Cecile; Berenshteyn, Frada; Lacoste, Arnaud; Thomas, Jason R; Roma, Guglielmo; et al. (2015). "SMN2 splice modulators enhance U1–pre-mRNA association and rescue SMA mice". Nature Chemical Biology. 11 (7): 511. doi:10.1038/nchembio.1837. PMID 26030728.
- ^ "LMI070". SMA News Today. Retrieved 2017-03-10.
- ^ "An Open Label Study of LMI070 in Type 1 Spinal Muscular Atrophy (SMA)". ClinicalTrials.gov. Retrieved 2017-03-10.