Amyotrophic lateral sclerosis: a neurodegenerative disorder poised for successful therapeutic translation.

ABSTRACT: Amyotrophic lateral sclerosis (ALS) is a devastating disease caused by degeneration of motor neurons. As with all major neurodegenerative disorders, development of disease-modifying therapies has proven challenging for multiple reasons. Nevertheless, ALS is one of the few neurodegenerative diseases for which disease-modifying therapies are approved. Significant discoveries and advances have been made in ALS preclinical models, genetics, pathology, biomarkers, imaging and clinical readouts over the last 10-15 years. At the same time, novel therapeutic paradigms are being applied in areas of high unmet medical need, including neurodegenerative disorders. These developments have evolved our knowledge base, allowing identification of targeted candidate therapies for ALS with diverse mechanisms

SUBMITTER: Mead RJ 

PROVIDER: S-EPMC9768794 | biostudies-literature | 2023 Mar

REPOSITORIES: biostudies-literature