Developing Novel Chemical Entities for the Treatment of Lysosomal Storage Disorders: An Academic Perspective.

Developing Novel Chemical Entities for the Treatment of Lysosomal Storage Disorders: An Academic Perspective. Am J Physiol Renal Physiol. 2015 Oct 7;:ajprenal.00393.2015 Authors: Shayman JA Abstract Historically, most FDA 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 start ups, small biotechnology companies, and academia for the identification of novel drug target 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 labs, 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. PMID: 26447223 [PubMed - as supplied by publisher]
Source: Am J Physiol Renal P... - Category: Urology & Nephrology Authors: Tags: Am J Physiol Renal Physiol Source Type: research