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BioWorld - Thursday, November 21, 2024
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» Disaster for Orphazyme as FDA rejects rare disease drug arimoclomol
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Disaster for Orphazyme as FDA rejects rare disease drug arimoclomol
June 18, 2021
By
Richard Staines
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Shares in Orphazyme A/S cratered after the FDA rejected its arimoclomol for Niemann-Pick disease type C, a rare and potentially fatal inherited condition where fat builds in tissues and organs.
BioWorld
Regulatory
Endocrine/metabolic
FDA