ABC transporter-driven pharmacoresistance in Amyotrophic Lateral Sclerosis.

ABSTRACT: Amyotrophic Lateral Sclerosis (ALS) is a slowly progressing neurodegenerative disease that affects motor neurons of the nervous system. Despite the identification of many potential therapeutics targeting pathogenic mechanisms in in vitro models, there has been limited progress in translating them into a successful pharmacotherapy in the animal model of ALS. Further, efforts to translate any promising results from preclinical trials to effective pharmacotherapies for patients have been unsuccessful, with the exception of riluzole, the only FDA-approved medication, which only modestly extends survival both in the animal model and in patients. Thus, it is essential to reconsider the strategies for developing ALS pharmacotherapies. Growing evidence suggests that problems identifying highly eff

SUBMITTER: Jablonski M 

PROVIDER: S-EPMC4344920 | biostudies-literature | 2015 May

REPOSITORIES: biostudies-literature