Arimoclomol maleate is primarily investigated for its potential therapeutic applications in treating neurodegenerative diseases and genetic disorders. It is being studied for its ability to enhance cellular stress responses, particularly in conditions like amyotrophic lateral sclerosis (ALS), Niemann-Pick disease type C, and Gaucher disease. The compound works by stabilizing heat shock proteins, which help in protein folding and cellular repair, making it a promising candidate for managing diseases caused by protein misfolding or aggregation. Clinical trials are ongoing to evaluate its efficacy and safety in these conditions.