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Clinical pharmacology considerations for first‐in‐human clinical trials for enzyme replacement therapy

13 May 2024

Abstract excerpt

Inborn errors of metabolism (IEM) such as lysosomal storage disorders (LSDs) are conditions caused by deficiency of one or more key enzymes, cofactors, or transporters involved in a specific metabolic pathway. Enzyme replacement therapy (ERT) provides an exogenous source of the affected enzyme and is one of the most effective treatment options for IEMs. In this paper, we review the first-in-human (FIH) protocols...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 38740427
DOI: 10.1002/jimd.12746

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