Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience.

@article{Zimran2010Phase1A,
  title={Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience.},
  author={Ari Zimran and Gheona Altarescu and Mici Philips and Drorit Attias and Marina Jmoudiak and Maher Deeb and Nan Wang and Kiran Bhirangi and Gabriel M. Cohn and Deborah Elstein},
  journal={Blood},
  year={2010},
  volume={115 23},
  pages={
          4651-6
        }
}
Enzyme replacement therapy is the standard of care for symptomatic Gaucher disease. Velaglucerase alfa is a human beta-glucocerebrosidase produced in a well-characterized human cell line. A 9-month phase 1/2 open-label, single-center trial and ongoing extension study were conducted to evaluate safety and efficacy of velaglucerase alfa. Twelve symptomatic adult type 1 Gaucher patients (intact spleens) received velaglucerase alfa (60 U/kg per infusion) during phase 1/2. An extension study was… CONTINUE READING

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