Novel small molecule TRVA242 targets neuromuscular junction in amyotrophic lateral sclerosis
Poulomee Bose
摘要：Research over the past decade has enabled a deeper understand-ing 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 pro-gression 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.
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资助基金：MITACS Elevate Post-Doctoral Fel-lowship co-funded by Treventis Inc. Toronto, Canada (CF134718/CF134719)
论文发表日期：2020-01-01
在线出版日期：2025-08-15（本平台首次上网日期，不代表文献的发表时间）
页数：2( 1041-1042 )