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Reductions in glucosylsphingosine (lyso-Gb1) in treatment-naïve and previously treated patients receiving velaglucerase alfa for type 1 Gaucher disease: Data from phase 3 clinical trials Deborah Elstein, Björn Mellgard, Quinn Dinh, Lan Lan, Yongchang Qiu, Claudia Cozma, Sabrina Eichler, Tobias Böttcher, Ari Zimran Molecular Genetics and Metabolism Volume 122, Issue 1, Pages (September 2017) DOI: /j.ymgme Copyright © 2017 The Authors Terms and Conditions
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Fig. 1 Overview of clinical trials. EOW, every other week.
Molecular Genetics and Metabolism , DOI: ( /j.ymgme ) Copyright © 2017 The Authors Terms and Conditions
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Fig. 2 Change in lyso-Gb1 concentrations during velaglucerase alfa treatment. (A) Treatment-naïve patients: all patients (n=22). (B) Treatment-naïve patients: percentage changes. (C) Switch patients: all patients (n=21). (D) Switch patients: percentage changes. Mean values are indicated by closed squares, median values are indicated by open circles. Molecular Genetics and Metabolism , DOI: ( /j.ymgme ) Copyright © 2017 The Authors Terms and Conditions
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Fig. 3 Impact of genotype on lyso-Gb1 concentrations and response during velaglucerase alfa treatment. (A) Treatment-naïve patients: observed concentrations. (B) Treatment-naïve patients: percentage changes. (C) Switch patients: observed concentrations. (D) Switch patients: percentage changes. Mean values are indicated by closed squares, median values are indicated by open circles. Molecular Genetics and Metabolism , DOI: ( /j.ymgme ) Copyright © 2017 The Authors Terms and Conditions
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Fig. 4 Spearman correlations between lyso-Gb1 concentrations and clinical disease parameters during velaglucerase alfa treatment. (A) treatment-naïve patients: platelet counts. (B) Treatment-naïve patients: spleen volume. (C) Switch patients: platelet counts. (D) Switch patients: spleen volume. MN, multiples of normal. Molecular Genetics and Metabolism , DOI: ( /j.ymgme ) Copyright © 2017 The Authors Terms and Conditions
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