The pharmacology of multiple regimens of agalsidase alfa enzyme replacement therapy for Fabry disease

@article{Clarke2007ThePO,
  title={The pharmacology of multiple regimens of agalsidase alfa enzyme replacement therapy for Fabry disease},
  author={Joe T. R. Clarke and Michael L. West and Jan Bultas and Raphael Schiffmann},
  journal={Genetics in Medicine},
  year={2007},
  volume={9},
  pages={504-509}
}
Purpose: This 10-week study was conducted to determine the pharmacokinetics of varying doses of agalsidase alfa and evaluate the effect of dose and dosing frequency on plasma Gb3 levels.Methods: Eighteen adult male Fabry patients, naive to enzyme replacement therapy, were randomized to one of five regimens: 0.1, 0.2, or 0.4 mg/kg weekly; 0.2 mg/kg every other week (the approved dose); or 0.4 mg/kg every other week. Intravenous infusion rate was 0.1 mg/kg per 20 minutes. Plasma Gb3 levels were… CONTINUE READING

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