"Best Practices for Development and Validation of Enzymatic Activity Assays to Support Drug Development for Inborn Errors of Metabolism and Biomarker Assessment"
Current treatments for inherited enzyme deficiencies include enzyme replacement therapy and small molecule chaperones, with gene and cell therapies showing promising potential. Enzyme activity assays play a crucial role in disease diagnosis, drug efficacy evaluation, and therapeutic development. This AAPS review explores the technical aspects of enzyme assays, including their design, validation, and life cycle management.
Read this article at: https://link.springer.com/article/10.1208/s12248-024-00966-y
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Pankajini Mallick Ph.D.
Sr. Principal Scientist, Preclinical Pharmacokinetics | Modeling and Simulation
San Diego CA
Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
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