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Enzyme therapy for the treatment of type 1 Gaucher disease: clinical outcomes and dose – response relationships

, MD PhD, , MD PhD, , MD, , MD & , PhD
Pages 2641-2652 | Published online: 10 Sep 2009

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Cristina Drugan, Tudor C. Drugan, Paula Grigorescu-Sido & Ioana Naşcu. (2017) Modelling long-term evolution of chitotriosidase in non-neuronopathic Gaucher disease. Scandinavian Journal of Clinical and Laboratory Investigation 77:4, pages 275-282.
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Cristina Drugan, Tudor C. Drugan, Nicolae Miron, Paula Grigorescu-Sido, Ioana Naşcu & Cristina Cătană. (2016) Evaluation of neopterin as a biomarker for the monitoring of Gaucher disease patients. Hematology 21:6, pages 379-386.
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Sawa Ito & A. John Barrett. (2013) Gauchers Disease—A Reappraisal of Hematopoietic Stem Cell Transplantation. Pediatric Hematology and Oncology 30:2, pages 61-70.
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Articles from other publishers (18)

Eline C.B. Eskes, Barbara Sjouke, Frédéric M. Vaz, Susan M.I. Goorden, André B.P. van Kuilenburg, Johannes M.F.G. Aerts & Carla E.M. Hollak. (2020) Biochemical and imaging parameters in acid sphingomyelinase deficiency: Potential utility as biomarkers. Molecular Genetics and Metabolism 130:1, pages 16-26.
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Juliette Berger, Marie Vigan, Bruno Pereira, Thu Thuy Nguyen, Roseline Froissart, Nadia Belmatoug, Florence Dalbiès, Agathe Masseau, Christian Rose, Christine Serratrice, Yves-Marie Pers, Ivan Bertchansky, Fabrice Camou, Monia Bengherbia, Céline Bourgne, Catherine Caillaud, Magali Pettazzoni, Amina Berrahal, Jérôme Stirnemann, France Mentré & Marc G. Berger. (2018) Intra-monocyte Pharmacokinetics of Imiglucerase Supports a Possible Personalized Management of Gaucher Disease Type 1. Clinical Pharmacokinetics 58:4, pages 469-482.
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. 2016. Diagnostic Imaging: Musculoskeletal Non-Traumatic Disease. Diagnostic Imaging: Musculoskeletal Non-Traumatic Disease 872 875 .
Neal J. Weinreb, David N. Finegold, Eleanor Feingold, Zhen Zeng, Barry E. Rosenbloom, Suma P. Shankar & Dominick Amato. (2015) Evaluation of disease burden and response to treatment in adults with type 1 gaucher disease using a validated disease severity scoring system (DS3). Orphanet Journal of Rare Diseases 10:1.
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Marta Moskot, Joanna Jakóbkiewicz-Banecka, Elwira Smolińska, Bogdan Banecki, Grzegorz Węgrzyn & Magdalena Gabig-Cimińska. (2015) Activities of genes controlling sphingolipid metabolism in human fibroblasts treated with flavonoids. Metabolic Brain Disease 30:5, pages 1257-1267.
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Amira Abdel Moneam Adly, Eman Abdel Rahman Ismail & Taghreed Mahmoud Ibraheem. (2015) Macrophage-derived soluble CD163 level in young patients with Gaucher disease: Relation to phenotypes, disease severity and complications. International Immunopharmacology 24:2, pages 416-422.
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Irena Žnidar, Tanya Collin-Histed, Pascal Niemeyer, Johanna Parkkinen, Anne-Grethe Lauridsen, Sandra Zariņa, Yossi Cohen & Jeremy Manuel. (2014) The European Gaucher Alliance: a survey of member patient organisations’ activities, healthcare environments and concerns. Orphanet Journal of Rare Diseases 9:1.
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L. van Dussen, E. J. Hendriks, J. E. M. Groener, R. G. Boot, C. E. M. Hollak & J. M. F. G. Aerts. (2014) Value of plasma chitotriosidase to assess non-neuronopathic Gaucher disease severity and progression in the era of enzyme replacement therapy. Journal of Inherited Metabolic Disease 37:6, pages 991-1001.
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Arndt Rolfs, Anne-Katrin Giese, Ulrike Grittner, Daniel Mascher, Deborah Elstein, Ari Zimran, Tobias Böttcher, Jan Lukas, Rayk Hübner, Uta Gölnitz, Anja Röhle, Ales Dudesek, Wolfgang Meyer, Matthias Wittstock & Hermann Mascher. (2013) Glucosylsphingosine Is a Highly Sensitive and Specific Biomarker for Primary Diagnostic and Follow-Up Monitoring in Gaucher Disease in a Non-Jewish, Caucasian Cohort of Gaucher Disease Patients. PLoS ONE 8:11, pages e79732.
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Simon Jones. 2013. Stem Cell Therapy in Lysosomal Storage Diseases. Stem Cell Therapy in Lysosomal Storage Diseases 21 35 .
Filippo Vairo, Cristina Netto, Alicia Dorneles, Suzana Mittelstadt, Matheus Wilke, Divair Doneda, Kristiane Michelin, Camila Blos Ribeiro, Amanda Quevedo, Tatiane Vieira, Tatiele Nalin, Sônia Lueska & Ida Vanessa D. Schwartz. 2013. JIMD Reports - Volume 11. JIMD Reports - Volume 11 1 6 .
Carla E. M. Hollak, Nadia Belmatoug, J. Alexander Cole, Stephan vom Dahl, Patrick B. Deegan, Jack Goldblatt, Barry Rosenbloom, Laura van Dussen, Anna Tylki‐Szymańska, Neal J. Weinreb, Ari Zimran & Maria Domenica Cappellini. (2012) Characteristics of type I Gaucher disease associated with persistent thrombocytopenia after treatment with imiglucerase for 4–5 years. British Journal of Haematology 158:4, pages 528-538.
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Ozlem Goker-Alpan. (2011) Therapeutic approaches to bone pathology in Gaucher disease: Past, present and future. Molecular Genetics and Metabolism 104:4, pages 438-447.
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Eman M Sherif, Azza AG Tantawy, Amira AM Adly, Hossam A Kader & Eman AR Ismail. (2011) D-dimer assay in Egyptian patients with Gaucher disease: correlation with bone and lung involvement. Blood Coagulation & Fibrinolysis 22:3, pages 176-184.
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Zahava R.S. Rosenberg-Yunger, Abdallah S. Daar, Halla Thorsteinsdóttir & Douglas K. Martin. (2011) Priority setting for orphan drugs: An international comparison. Health Policy 100:1, pages 25-34.
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Stephan vom Dahl & Eugen Mengel. (2010) Lysosomal storage diseases as differential diagnosis of hepatosplenomegaly. Best Practice & Research Clinical Gastroenterology 24:5, pages 619-628.
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You-Hai Xu, Ying Sun, Sonya Barnes & Gregory A. Grabowski. (2010) Comparative Therapeutic Effects of Velaglucerase Alfa and Imiglucerase in a Gaucher Disease Mouse Model. PLoS ONE 5:5, pages e10750.
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M.H. de Ru, F.A. Wijburg, J.M.F.G. Aerts, E.D.M. Post & C.E.M. Hollak. (2012) Anemie, trombocytopenie en hepatosplenomegalie: de ziekte van Gaucher. Tijdschrift voor Kindergeneeskunde 78:2, pages 73-77.
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