19561-95-6Relevant academic research and scientific papers
Novel compounds that reverse the disease phenotype in Type 2 Gaucher disease patient-derived cells
Abou-Gharbia, Magid,Childers, Wayne,Colussi, Dennis J.,Fan, Rong,Gordon, John,Jacobson, Marlene A.,Liu, Yuxiao,Martinez, Rogelio,Melenski, Edward
supporting information, (2019/12/11)
Gaucher disease (GD) results from inherited mutations in the lysosomal enzyme β-glucocerobrosidase (GCase). Currently available treatment options for Type 1 GD are not efficacious for treating neuronopathic Type 2 and 3 GD due to their inability to cross the blood-brain barrier. In an effort to identify small molecules which could be optimized for CNS penetration we identified tamoxifen from a high throughput phenotypic screen on Type 2 GD patient-derived fibroblasts which reversed the disease phenotype. Structure activity studies around this scaffold led to novel molecules that displayed improved potency, efficacy and reduced estrogenic/antiestrogenic activity compared to the original hits. Here we present the design, synthesis and structure activity relationships that led to the lead molecule Compound 31.
NOVEL FUNCTIONALIZED N,N-DIALKYLAMINO PHENYL ETHERS AND THEIR METHOD OF USE
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Paragraph 0276; 0277, (2017/12/29)
Pharmaceutical compositions of the invention comprise functionalized N,N-dialkylamino phenyl ethers derivatives having a disease-modifying action in the treatment of diseases associated with lysosomal storage dysfunction that include Gaucher's disease, and any disease or condition involving lysosomal storage dysfunction.
Triphenylalkene derivatives and their use as selective estrogen receptor modulators
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, (2008/06/13)
The invention provides novel selective estrogen receptor modulator compounds of the general formula: wherein R1 and R2, which are the same or different are a) H, halogen, OCH3, OH; or ?where X is O, NH or S; and n is an integer from 1 to 4; and R4 and R5, which are the same or different, are a 1 to 4 carbon alkyl, H, —CH2C≡CH or —CH2CH2OH; or R4 and R5 form an N-containing five- or six-membered ring or heteroaromatic ring; or c) —Y—(CH2)nCH2—O—R6 where Y is O, NH or S and n is an integer from 1 to 4; and R6 is H, —CH2CH2OH, or —CH2CH2Cl; or d) 2,3-dihydroxypropoxy, 2-methylsulfamylethoxy, 2-chloroethoxy, 1-ethyl-2-hydroxyethoxy, 2,2-diethyl-2-hydroxyethoxy or carboxymethoxy; and R3 is H, halogen, OH or —OCH3; stereoisomers thereof and their non-toxic pharmaceutically acceptable salts and esters and mixtures thereof, which compounds exhibit valuable pharmacological properties.
Synthesis of -tamoxifen; A Mechanistic Probe of Tamoxifen Induced Hepatic DNA Adduct Formation
Horton, Martin N.,Jarman, Michael,Potter, Gerard A.
, p. 767 - 772 (2007/10/02)
Tamoxifen which incorporates a fully deuterated ethyl group, 5-ethyl>-tamoxifen, has been synthesised in order to probe the mechanism of tamoxifen induced hepatic DNA adduct formation.The pentadeuteroethyl group was introduced into the tamoxi
