Population Pharmacokinetic/Pharmacodynamic Modeling of Therapeutic Enzymes in Lysosomal Storage Diseases
Ina Barzel, Ans T. van der Ploeg, W. W. M. Pim Pijnappel, P. Hugo M. van der Kuy, Johanna M. P. van den Hout, Tim Preijers · 2026
Read Population Pharmacokinetic/Pharmacodynamic Modeling of Therapeutic Enzymes in Lysosomal Storage Diseases by Ina Barzel, Ans T. van der Ploeg, W. W. M. Pim Pijnappel, P. Hugo M. van der Kuy, Johanna M. P. van den Hout, Tim Preijers (2026) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/pmc-13183719.txt.
Description
Children and adults with lysosomal storage diseases get enzyme infusions, but doses are hard to set because patients respond so unevenly. Ina Barzel and co-authors review the math models built to guide that dosing in this 2026 paper. They gathered every published population model of these enzymes, nine in all, and compared how each was built and checked.
Most models describe the drug's path through the body well, splitting it into two or three compartments. Body weight stood out as the main factor shifting drug levels between patients. Models of the drug's effect, as opposed to its movement, remain scarce and simple. The authors point to richer effect models as the clear next step for the field.
Catalog record
| CREATOR | Ina Barzel, Ans T. van der Ploeg, W. W. M. Pim Pijnappel, P. Hugo M. van der Kuy, Johanna M. P. van den Hout, Tim Preijers |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 76 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |