Novel small molecule TRVA242 targets neuromuscular junction in amyotrophic lateral sclerosis

作     者：Poulomee Bose Poulomee Bose

作者机构：Department of Neuroscience Centre Hospitalier Universitaire St. Justine Université de Montréal 

出 版 物：《Neural Regeneration Research》 (中国神经再生研究（英文版）)

年 卷 期：2020年第15卷第6期

页      面：1041-1042页

核心收录：

学科分类：0710[理学-生物学] 1002[医学-临床医学] 1001[医学-基础医学(可授医学、理学学位)] 10[医学] 

基　　金：supported by MITACS Elevate Post-Doctoral Fellowship co-funded by Treventis Inc.Toronto Canada(CF134718/CF134719) 

主　　题：amyotrophic sclerosis mostly 

摘      要：Research over the past decade has enabled a deeper understanding of the pathophysiology of amyotrophic lateral sclerosis(ALS).While 10%of all ALS cases have been reported to be familial with a clear Mendelian inheritance,clinically,sporadic and familial forms of ALS cannot be distinguished(Robberecht and Philips,2013).Presently there are only two Food and Drug Administration approved treatment options for ALS-riluzole and radicava(also known as edavarone).Riluzole is mostly known to delay the onset of ventilator dependence and extends the life span by 2–3 months;edavarone on the other hand has been reported slow disease progression at all stages in ALS(Jaiswal,2019).However,given the multifaceted nature of ALS,there is an urgent need to identify more molecules with a strong therapeutic potential.