What Is in the Literature

J Clin Neuromuscul Dis. 2019 Dec;21(2):84-89. doi: 10.1097/CND.0000000000000249.

Abstract

This installment of what is in the literature is on amyotrophic lateral sclerosis (ALS). The pathophysiology of ALS remains open and the role of genes, a foothold into pathophysiology, but there are >22 genes identified, and the mechanisms are not known for any. Despite the lack of a firm understanding of pathophysiology, drug trials continue based on possible mechanisms, but no new drugs beyond riluzole and edaravone have been positive in phase 3 trials. There are a number of formal stem cell trials underway, and the results of a phase 2 trial are described. Major efforts to make trials more sensitive are being considered. There are a number of articles with helpful and practical findings for the diagnosis and management of ALS.

Publication types

  • Review

MeSH terms

  • Amyotrophic Lateral Sclerosis / drug therapy
  • Amyotrophic Lateral Sclerosis / genetics
  • Amyotrophic Lateral Sclerosis / physiopathology*
  • Amyotrophic Lateral Sclerosis / therapy
  • Clinical Trials as Topic
  • Humans
  • Neuroprotective Agents
  • Stem Cell Transplantation

Substances

  • Neuroprotective Agents