| Autor: |
Darwish, Mona, Marbury, Thomas C., Nunez, Rene, Youakim, James M., An, Di, Darling, Inger, Lukacova, Viera, Bishop, Kathie M. |
| Zdroj: |
European Journal of Drug Metabolism and Pharmacokinetics; 20240101, Issue: Preprints p1-16, 16p |
| Abstrakt: |
Trofinetide is the first approved treatment for Rett syndrome, a rare genetic condition that affects brain development. People with Rett syndrome do not usually experience problems with their kidneys but, because trofinetide is mostly removed from the body in the urine, it is important to check if the dose needs to be reduced when the kidneys do not work properly. Researchers first used computer modeling to predict how much the recommended dose of trofinetide in people with normal kidney function would have to be reduced in virtual populations with mild, moderate, or severe kidney disease, or kidney failure. To check if the computer model was correct, the blood levels of trofinetide were compared between 10 people with moderate kidney disease given a 50% reduced dose as predicted by the model and 10 healthy people given the recommended dose. The levels of trofinetide measured in people with moderate kidney disease were similar to the levels in healthy people and were similar to the predicted levels in the model, so together these results supported the 50% dose reduction in people with moderate kidney disease. The similarity between the results in the study and the model confirms that the model is good at predicting how much the dose needs to change in people with kidney disease. |
| Databáze: |
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