Orphazyme A/S
USE OF ARIMOCLOMOL IN THE TREATMENT OF NIEMANN PICK DISEASE

Abstract:

This application is directed to assays, diagnostic methods and treatment methods using arimoclomol for the treatment of Niemann-Pick disease type C (NPC). More specifically, this application is directed to assays, diagnostics methods and treatment methods that target specific clinical subsets of patients with NPC who exhibit at least one missense mutation in at least one allele of an NPC gene.