Orphazyme A/S, formerly Orphazyme ApS, is a Denmark-based company active within the biopharmaceutical industry. It develops new therapies for the treatment of a family of genetic disorders. The Company's main focus is on preclinical development of medicines for the treatment of lysosomal storage diseases (LSDs), a group of approximately 50 genetic disorders that result from deficiencies of lysosomal enzymes or other lysosomal components, and protein aggregation diseases. The Company’s candidate, arimoclomol, stimulates the production of the cells’ protein rescue system, the heat shock proteins, which helps the misfolded proteins to either get back to their functional shape or, if this cannot be achieved, remove them from the cells by means of the cells’ recycling system, the lysosomes, so they no longer form toxic aggregates. The Company collaborates with academic institutions in Europe and the United States.
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