Neural Regeneration Research ›› 2020, Vol. 15 ›› Issue (6): 1041-1042.doi: 10.4103/1673-5374.270309

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Novel small molecule TRVA242 targets neuromuscular junction in amyotrophic lateral sclerosis

Poulomee Bose   

  1. Department of Neuroscience, Centre Hospitalier Universitaire St. Justine, Université de Montréal, Montréal, QC, Canada
  • Online:2020-06-15 Published:2020-07-02
  • Contact: Poulomee Bose, PhD,poulomee.bose@umontreal.ca.
  • Supported by:
    This work was supported by MITACS Elevate Post-Doctoral Fellowship co-funded by Treventis Inc. Toronto, Canada (CF134718/ CF134719).

Abstract: 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.