Therapeutic targeting of ALS pathways: Refocusing an incomplete picture.

ABSTRACT: Numerous potential amyotrophic lateral sclerosis (ALS)-relevant pathways have been hypothesized and studied preclinically, with subsequent translation to clinical trial. However, few successes have been observed with only modest effects. Along with an improved but incomplete understanding of ALS as a neurodegenerative disease is the evolution of more sophisticated and diverse in vitro and in vivo preclinical modeling platforms, as well as clinical trial designs. We highlight proposed pathological pathways that have been major therapeutic targets for investigational compounds. It is likely that the failures of so many of these therapeutic compounds may not have occurred because of lack of efficacy but rather because of a lack of preclinical modeling that would help define an appropriate dis

SUBMITTER: Maragakis NJ 

PROVIDER: S-EPMC10647018 | biostudies-literature | 2023 Nov

REPOSITORIES: biostudies-literature