Novel small molecule TRVA242 targets neuromuscular junction in amyotrophic lateral sclerosis

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.