Low-dose enzyme replacement therapy for Gaucher's disease: effects of age, sex, genotype, and clinical features on response to treatment.

@article{Zimran1994LowdoseER,
  title={Low-dose enzyme replacement therapy for Gaucher's disease: effects of age, sex, genotype, and clinical features on response to treatment.},
  author={Ari Zimran and Deborah Elstein and Ruth Kannai and Shoshana Zevin and Irith Hadas-Halpern and Ephrat Levy-Lahad and Yves S Cohen and Michael Horowitz and Aya Abrahamov},
  journal={The American journal of medicine},
  year={1994},
  volume={97 1},
  pages={3-13}
}
Although alglucerase therapy has become the treatment of choice for symptomatic patients with Gaucher's disease, the low-dose/high-frequency regimen introduced as a means to reduce the high cost of treatment has raised major controversy. We evaluated the efficacy and safety of low-dose alglucerase in 29 patients with Gaucher's disease who completed 6 to 28 months of therapy. All received intravenous alglucerase at a monthly dose of 30 units/kg, given usually in equal doses 3 times a week. All… CONTINUE READING

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