ARIMOCLOMOL – HARNESSING THE CELL-PROTECTIVE PROPERTIES OF THE HEAT-SHOCK RESPONSE

Arimoclomol is designed to selectively amplify the natural role of endogenous heat-shock proteins, which essentially act as the cells' lifeguards, protecting them against toxicity caused by protein misfolding, aggregation and lysosomal dysfunction. Arimoclomol was sold to Orphazyme A/S and is currently in development in four diseases, including Niemann Pick disease Type C (NPC), Amyotrophic Lateral Sclerosis (ALS), Inclusion Body Myositis (IBM) and Gaucher disease. The aim is to slow or even prevent disease progression and Orphazyme believes their approach has the potential to dramatically improve the lives of those suffering from a range of neurodegenerative orphan diseases, which currently do not have satisfactory treatments.