SynthesisClinical pharmacokinetics2026
Population Pharmacokinetic/Pharmacodynamic Modeling of Therapeutic Enzymes in Lysosomal Storage Diseases.
Synthesis in Clinical pharmacokinetics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundLysosomal 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.
objectivesThis review aimed to provide a comprehensive overview of published popPK and popPD models of therapeutic enzymes in LSDs, summarize modeling strategies and study characteristics, and evaluate the quality of the available models.
methodsA systematic search of Medline, Embase, and Web of Science (inception-March 2025) identified studies reporting popPK and/or popPD models of therapeutic enzymes in patients with LSDs. Data on study characteristics, structural and statistical model choices, covariate analyses, and evaluation methods were extracted and compared.
resultsThis review included studies describing 6 popPK models and 3 popPD models of therapeutic enzymes in LSD. All models were developed using the nonlinear mixed-effects (NLME) modeling approach. The PK characteristics were adequately described using a two-compartment model in three studies, a three-compartment model in two studies, and a one-compartment model in one study. Three studies additionally assessed PK parameters in monocytes, leukocytes, and cerebrospinal fluid (CSF). Among all tested covariates, total body weight (TBW) was identified as a significant predictor for clearance (CL) and volume of distribution in the central compartment (V
conclusionsPopPK modeling of therapeutic enzymes in LSDs is relatively well established. However, progress in popPD modeling remains limited. Existing models support the use of indirect response and maximum effect (E
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