1424-74-4Relevant articles and documents
Discovery and Optimization of Small Molecule Splicing Modifiers of Survival Motor Neuron 2 as a Treatment for Spinal Muscular Atrophy
Woll, Matthew G.,Qi, Hongyan,Turpoff, Anthony,Zhang, Nanjing,Zhang, Xiaoyan,Chen, Guangming,Li, Chunshi,Huang, Song,Yang, Tianle,Moon, Young-Choon,Lee, Chang-Sun,Choi, Soongyu,Almstead, Neil G.,Naryshkin, Nikolai A.,Dakka, Amal,Narasimhan, Jana,Gabbeta, Vijayalakshmi,Welch, Ellen,Zhao, Xin,Risher, Nicole,Sheedy, Josephine,Weetall, Marla,Karp, Gary M.
supporting information, p. 6070 - 6085 (2016/07/26)
The underlying cause of spinal muscular atrophy (SMA) is a deficiency of the survival motor neuron (SMN) protein. Starting from hits identified in a high-throughput screening campaign and through structure-activity relationship investigations, we have developed small molecules that potently shift the alternative splicing of the SMN2 exon 7, resulting in increased production of the full-length SMN mRNA and protein. Three novel chemical series, represented by compounds 9, 14, and 20, have been optimized to increase the level of SMN protein by >50% in SMA patient-derived fibroblasts at concentrations of 160 nM. Daily administration of these compounds to severe SMA Δ7 mice results in an increased production of SMN protein in disease-relevant tissues and a significant increase in median survival time in a dose-dependent manner. Our work supports the development of an orally administered small molecule for the treatment of patients with SMA.
Development of a practical and scalable synthesis of a potent CRTH2 antagonist
Yoshida, Shinya,Yoshino, Toshitaka,Miyafuji, Akio,Yasuda, Hironobu,Kimura, Takenori,Takahashi, Takumi,Mukuta, Takashi
, p. 1544 - 1551 (2013/02/23)
This contribution describes the process research and development of a practical and scalable synthetic method towards compound 1, which has a potent CRTH2 antagonistic activity. The medicinal chemistry synthetic route and second generation synthetic route
COMPOUNDS AND COMPOSITIONS AS MODULATORS OF GPR119 ACTIVITY
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Page/Page column 154-155, (2008/12/08)
The invention provides compounds, pharmaceutical compositions comprising such compounds and methods of using such compounds to treat or prevent diseases or disorders associated with the activity of GPR119.
Studies on the hydrogenolysis of benzyl ethers
Llàcer, Enric,Romea, Pedro,Urpí, Fèlix
, p. 5815 - 5818 (2007/10/03)
Selective hydrogenolysis of benzyl ethers can be achieved by the appropriate choice of the catalyst, solvent, and concentration, which facilitates the design of O-benzyl based protecting group strategies for the synthesis of natural products.
Compounds and their use in medicine, process for their preparation and pharmaceutical compositions containing them
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Page 20, (2008/06/13)
The present invention relates to novel antidiabetic, hypolipidemic, antiobesity and hypocholesterolemic compounds of formula (I), their derivatives, their analogs, their tautomeric forms, their stereoisomers, their polymorphs, their pharmaceutically acceptable salts, their pharmaceutically acceptable solvates and pharmaceutically acceptable compositions containing them.
Aryl carboxylic acid and aryl tetrazole derivatives as IP receptor modulators
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Example 11, (2010/02/04)
This invention relates to compounds which are generally IP receptor modulators, particularly IP receptor agonists, and which are represented by Formula I: wherein R1, R2, R3, R4, R5, A, and B are as defined in the specification, and individual isomers, racemic or non-racemic mixtures of isomers, and pharmaceutically acceptable salts or solvates thereof. The invention further relates to pharmaceutical compositions containing such compounds and methods for their use as therapeutic agents.
Lipase-catalysed chemoselective monoacetylation of hydroxyalkylphenols and chemoselective removal of a single acetyl group from their diacetates
Allevi, Pietro,Ciuffreda, Pierangela,Longo, Alessandra,Anastasia, Mario
, p. 2915 - 2924 (2007/10/03)
It was demonstrated that Pseudomonas cepacia PS lipase adsorbed on Celite, has the ability to catalyse the chemoselective monoacetylation of various hydroxyalkylphenols or the chemoselective removal of a single acetyl group from the corresponding acetate.
Synthesis and biological activity of new 3-hydroxy-3-methylglutaryl-CoA synthase inhibitors: 2-Oxetanones with a meta-substituent on the benzene ring in the side chain
Hashizume,Ito,Kanaya,Nagashima,Usui,Oshima,Kanao,Tomoda,Sunazuka,Kumagai,Omura
, p. 1272 - 1278 (2007/10/02)
Isosteric side chain analogs of 3a were synthesized and tested for inhibitory activities towards 3-hydroxy-3-methylglutaryl coenzyme A (HMG- CoA) synthase and upon cholesterol production in Hep G2 cells and in mouse liver. It became clear that the lipophi
