Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double-blind, multinational, Phase 3 study.

@article{Gonzalez2013EnzymeRT,
  title={Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double-blind, multinational, Phase 3 study.},
  author={Derlis E Gonzalez and Hadhami Ben Turkia and Elena A Lukina and Isaac Kisinovsky and Marie-Françoise Ben Dridi and Deborah Elstein and David Zahrieh and Eric Crombez and Kiran Bhirangi and Norman W. Barton and Ari Zimran},
  journal={American journal of hematology},
  year={2013},
  volume={88 3},
  pages={166-71}
}
Type 1 Gaucher disease (GD1), resulting from glucocerebrosidase deficiency, leads to splenomegaly, hepatomegaly, anemia, thrombocytopenia, and bone involvement. Current standard treatment is enzyme replacement therapy. Velaglucerase alfa is an enzyme replacement product for GD1, with the same amino acid sequence as naturally occurring human glucocerebrosidase. This multinational, Phase 3 trial evaluated the efficacy and safety of two doses of velaglucerase alfa in 25 treatment-naïve, anemic… CONTINUE READING

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