| 词条 | Branaplam |
| 释义 |
| IUPAC_name = (6E)-3-(1H-pyrazol-4-yl)-6-[3-(2,2,6,6-tetramethylpiperidin-4-yl)oxy-1H-pyridazin-6-ylidene]cyclohexa-2,4-dien-1-one | image = Branaplam skeletal.svg | alt = | caption = | pronounce = | tradename = | Drugs.com = | MedlinePlus = | pregnancy_AU = | pregnancy_AU_comment = | pregnancy_US = | pregnancy_category= | routes_of_administration = | legal_AU = | legal_AU_comment = | legal_CA = | legal_DE = | legal_NZ = | legal_UK = | legal_US = | legal_UN = | legal_status = | bioavailability = | protein_bound = | metabolism = | metabolites = | onset = | elimination_half-life = | duration_of_action = | excretion = | CAS_number = 1562338-42-4 | class = | ATCvet = | ATC_prefix = | ATC_suffix = | PubChem = 89971189 | UNII_Ref = | UNII = P12R69543A | DrugBank = | ChemSpiderID = 34980709 | synonyms = LMI070; NVS-SM1 | C=22 | H=27 | N=5 | O=2 | molecular_weight = 393.491 g/mol | smiles = CC1(CC(CC(N1)(C)C)Oc2ccc(nn2)c3ccc(cc3O)c4c[nH]nc4)C | 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+ | StdInChIKey = YIFFDXMJVNKGBL-ISLYRVAYSA-N | StdInChI2 = 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) | StdInChIKey2 = STWTUEAWRAIWJG-UHFFFAOYSA-N }}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 is a pyridazine derivative that 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|2017|03}}, branaplam is in a phase-II clinical trial in children with SMA type 1.[3][4]{{clear left}} References1. ^{{cite journal| doi=10.1038/nchembio.1837| pmid=26030728| title=SMN2 splice modulators enhance U1–pre-mRNA association and rescue SMA mice| journal=Nature Chemical Biology| volume=11| issue=7| pages=511| year=2015| last1=Palacino| first1=James| last2=Swalley| first2=Susanne E| last3=Song| first3=Cheng| last4=Cheung| first4=Atwood K| last5=Shu| first5=Lei| last6=Zhang| first6=Xiaolu| last7=Van Hoosear| first7=Mailin| last8=Shin| first8=Youngah| last9=Chin| first9=Donovan N| last10=Keller| first10=Caroline Gubser| last11=Beibel| first11=Martin| last12=Renaud| first12=Nicole A| last13=Smith| first13=Thomas M| last14=Salcius| first14=Michael| last15=Shi| first15=Xiaoying| last16=Hild| first16=Marc| last17=Servais| first17=Rebecca| last18=Jain| first18=Monish| last19=Deng| first19=Lin| last20=Bullock| first20=Caroline| last21=McLellan| first21=Michael| last22=Schuierer| first22=Sven| last23=Murphy| first23=Leo| last24=Blommers| first24=Marcel J J| last25=Blaustein| first25=Cecile| last26=Berenshteyn| first26=Frada| last27=Lacoste| first27=Arnaud| last28=Thomas| first28=Jason R| last29=Roma| first29=Guglielmo| last30=Michaud| first30=Gregory A| display-authors=29}} {{drug-stub}}2. ^{{cite web | url=https://smanewstoday.com/lmi070-novartis/ | title=LMI070 | publisher=SMA News Today | accessdate=2017-03-10}} 3. ^{{cite web| url=https://clinicaltrials.gov/ct2/show/NCT02268552 |title=An Open Label Study of LMI070 in Type 1 Spinal Muscular Atrophy (SMA) | publisher=ClinicalTrials.gov | accessdate=2017-03-10}} 4. ^{{cite web | url=http://www.curesma.org/news/novartis-branaplam-update.html | title=Novartis Releases Update on LMI070 (Branaplam) Clinical Trial | publisher = CureSMA | date = 2017-09-20 | accessdate=2017-10-07 }} 4 : Spinal muscular atrophy|Experimental drugs|Novartis|Novartis brands |
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