title: Developing novel chemical entities for the treatment of lysosomal storage disorders: an academic perspective.
abstract: Historically, most Federal Drug Administration-approved drugs were the result of "in-house" efforts within large pharmaceutical companies. Over the last two decades, this paradigm has steadily shifted as the drug industry turned to startups, small biotechnology companies, and academia for the identification of novel drug targets and early drug candidates. This strategic pivot has created new opportunities for groups less traditionally associated with the creation of novel therapeutics, including small academic laboratories, for engagement in the drug discovery process. A recent example of the successful development of a drug that had its origins in academia is eliglustat tartrate, an oral agent for Gaucher disease type 1.
authors.affiliation: Department of Internal Medicine, University of Michigan Medical School, University of Michigan, Ann Arbor, Michigan jshayman@umich.edu.
authors.forename: James A
authors.initials: JA
authors.lastname: Shayman
mesh_terms: D000818:Animals; D055808:Drug Discovery / Q000639:trends*; D004791:Enzyme Inhibitors / Q000627:therapeutic use*; D005776:Gaucher Disease / Q000188:drug therapy*; D006801:Humans; D007221:Industry / Q000639:trends*; D016464:Lysosomal Storage Diseases / Q000188:drug therapy*; D012106:Research / Q000639:trends*
chemical_list: D004791:Enzyme Inhibitors
keywords: Gaucher disease; eliglustat tartrate; glucosylceramide; glucosylceramide synthase; lysosomal storage disease