{"id":11381,"date":"2022-11-24T03:06:45","date_gmt":"2022-11-23T19:06:45","guid":{"rendered":"https:\/\/www.pyrazoles-derivatives.com\/?p=11381"},"modified":"2022-11-24T03:06:45","modified_gmt":"2022-11-23T19:06:45","slug":"cheung-atwood-k-s-team-published-research-in-journal-of-medicinal-chemistry-in-61-cas-763120-58-7","status":"publish","type":"post","link":"https:\/\/www.pyrazoles-derivatives.com\/?p=11381","title":{"rendered":"Cheung, Atwood K.&#8217;s team published research in Journal of Medicinal Chemistry  in 61 | CAS: 763120-58-7"},"content":{"rendered":"<p>Cheung, Atwood K. published the artcile<b><i>Discovery of Small Molecule Splicing Modulators of Survival Motor Neuron-2 (SMN2) for the Treatment of Spinal Muscular Atrophy (SMA)<\/i><\/b>,  <a href=\"https:\/\/www.ambeed.com\/products\/763120-58-7.html\">HPLC of Formula: 763120-58-7<\/a>,  the publication is  Journal of Medicinal Chemistry (2018), 61(24), 11021-11036, database is CAplus and MEDLINE.<\/p>\n<p>Spinal muscular atrophy (SMA), a rare neuromuscular disorder, is the leading genetic cause of death in infants and toddlers. SMA is caused by the deletion or a loss of function mutation of the survival motor neuron 1 (SMN1) gene. In humans, a second closely related gene SMN2 exists, however it codes for a less stable SMN protein. In recent years, significant progress has been made toward disease modifying treatments for SMA by modulating SMN2 pre-mRNA splicing. Herein, we describe the discovery of LMI070\/branaplam, a small mol. that stabilizes the interaction between the spliceosome and SMN2 pre-mRNA. Branaplam (1) originated from a high-throughput phenotypic screening hit, pyridazine 2, and evolved via multi-parameter lead optimization. In a severe mouse SMA model, branaplam treatment increased full-length SMN RNA and protein levels, and extended survival. Currently, branaplam is in clin. studies for SMA.<\/p>\n<p>Journal of Medicinal Chemistry published new progress about 763120-58-7. 763120-58-7 belongs to pyrazoles-derivatives, auxiliary class Pyrazole,Boronic acid and ester,Boronic Acids,Boronic acid and ester, name is 1H-Pyrazole-4-boronic acid, and the molecular formula is C3H5BN2O2, <a href=\"https:\/\/www.ambeed.com\/products\/763120-58-7.html\">HPLC of Formula: 763120-58-7<\/a>.<\/p>\n<p>Referemce:<br \/><a href=\"https:\/\/en.wikipedia.org\/wiki\/Pyrazole\">https:\/\/en.wikipedia.org\/wiki\/Pyrazole<\/a>,<br \/><a href=\"Pyrazole - Wikipedia\">Pyrazoles &#8211; an overview | ScienceDirect Topics<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Journal of Medicinal Chemistry published new progress about 763120-58-7. 763120-58-7 belongs to pyrazoles-derivatives, auxiliary class Pyrazole,Boronic acid and ester,Boronic Acids,Boronic acid and ester, name is 1H-Pyrazole-4-boronic acid, and the molecular formula is C3H5BN2O2, <a href=\"https:\/\/www.ambeed.com\/products\/763120-58-7.html\">HPLC of Formula: 763120-58-7<\/a>.<\/p>\n","protected":false},"author":8,"featured_media":0,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[802,131],"tags":[732],"class_list":["post-11381","post","type-post","status-publish","format-standard","hentry","category-763120-58-7","category-pyrazoles-derivatives","tag-m-w100-150"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v24.9 - 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