Population Pharmacokinetic/Pharmacodynamic Modeling of Therapeutic Enzymes in Lysosomal Storage Diseases.
Barzel I, van der Ploeg AT, Pijnappel WWMP, van der Kuy PHM, van den Hout JMP, Preijers T
Clinical pharmacokinetics, 2026 May 08
Abstract
Lysosomal storage diseases (LSDs) are rare genetic disorders treated with enzyme replacement therapy (ERT). However, treatment outcomes are highly variable, reflecting the complexities of therapeutic enzyme pharmacology, patient heterogeneity and therapy response. Population pharmacokinetic/pharmacodynamic (popPK/PD) modeling can help characterize this variability, identify covariates, and optimize dosing strategies.