11 minute read
May 7, 2022

Review of 2021 FDA-Approved Drugs for Genetic Conditions

Drug Hunter Team

The FDA approved five new drugs in 2021 for use for genetic conditions, including molybdenum cofactor deficiency, Von Hippel-Lindau (VHL) disease, achondroplasia, and progressive familial intrahepatic cholestasis (PFIC). This article and slide deck reviews each in detail, including their mechanisms of action, originators, key clinical data, disease context, target rationale, and more…



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