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 by amplifying the heat shock response, which increases the expression of heat shock proteins (HSPs) specifically under stress conditions. These HSPs help in protein folding and cellular repair, making it a promising candidate for managing diseases caused by protein misfolding or aggregation, such as amyotrophic lateral sclerosis (ALS), Niemann-Pick disease type C, and Gaucher disease. Clinical trials are ongoing to evaluate its efficacy and safety in these conditions.