中国神经再生研究(英文版) ›› 2020, Vol. 15 ›› Issue (6): 1041-1042.doi: 10.4103/1673-5374.270309

• 观点:退行性病与再生 • 上一篇    下一篇

用于神经肌肉接头的小分子靶向治疗肌萎缩侧索硬化症

  

  • 出版日期:2020-06-15 发布日期:2020-07-02

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).

摘要: orcid: 0000-0002-8947-0118 (Poulomee Bose)

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.