Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4 years of treatment.

@article{Lukina2014EliglustatAI,
  title={Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4 years of treatment.},
  author={Elena Lukina and Nora Patricia Watman and Marta Dragosky and Gregory McCarthy Pastores and Elsa Avila Arreguin and Hanna Rosenbaum and Ari Zimran and Jennifer Angell and Leorah Ross and Ana Cristina Puga and Judith M Peterschmitt},
  journal={Blood cells, molecules & diseases},
  year={2014},
  volume={53 4},
  pages={274-6}
}
Eliglustat is an investigational, oral substrate reduction therapy for Gaucher disease type 1 (GD1). Nineteen treatment-naïve patients have now completed 4years of an open-label study (NCT00358150). Mean hemoglobin level and platelet count increased by 2.3±1.5g/dL (baseline: 11.3±1.5g/dL) and 95% (baseline: 68,700±21,200/mm(3)), respectively. Mean spleen and liver volumes (multiples of normal, MN) decreased by 63% (baseline: 17.3±9.5 MN) and 28% (baseline: 1.7±0.4 MN), respectively. Median… CONTINUE READING

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Eliglustat: A Review in Gaucher Disease Type 1

Drugs • 2015
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Pharmacotherapy of Gaucher Disease: Current and Future Options.

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