Orphazyme A/S is a biopharmaceutical company that develops therapies for the treatment of neurodegenerative orphan diseases. The Company focuses on the amplification of heat-shock proteins to develop and commercialize therapeutics for diseases caused by protein misfolding and aggregation, and lysosomal dysfunction, including lysosomal storage and neuromuscular degenerative diseases. Orphazyme conducts its U.S. operations through its whollyowned subsidiary, Orphazyme US, which is focused on U.S. regulatory review and preparing for the Company’s first potential U.S. commercial launch, including legal, commercial, finance, advocacy relations, regulatory, and medical affairs functions. Orphazyme’s lead drug candidate is arimoclomol, which is in clinical development for four orphan diseases, including Niemann-Pick disease type C (“NPC”), Amyotrophic Lateral Sclerosis (“ALS”), and Inclusion Body Myositis (“IBM”).
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