Orphazyme A/S is a biopharmaceutical entity focused on developing therapeutic interventions for rare neurological conditions. The company's strategy involves amplifying heat-shock proteins to create and commercialize treatments for diseases characterized by protein misfolding and aggregation, as well as lysosomal impairments, which encompass lysosomal storage and neuromuscular degenerative disorders. Their leading experimental drug, Arimoclomol, is currently in clinical development, being investigated for its potential in four distinct orphan diseases: Niemann-Pick disease type C, Amyotrophic Lateral Sclerosis (ALS), Inclusion Body Myositis, and Gaucher disease.