Arimoclomol

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 KemPharm A/S and is currently in development in Niemann Pick disease Type C (NPC) and Gaucher disease. The aim is to slow or even prevent disease progression and KemPharm 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.