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Agalsidase Beta Clinical Trials and Long Term Experience

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Abstract

There have been phase I/II and III clinical trials of agalsidase beta infusions for treatment of Fabry disease. The phase I/II trial demonstrated that agalsidase beta infusions were well tolerated, and that globotriaocylceramide (GL-3) clearance was dose dependent. A phase III trials demonstrated clearance of GL-3 on a tissue level. A separate study demonstrated decreased risk of renal progression with therapy in a population with advanced disease. There is evidence of benefit for the renal, cardiac, and quality of life manifestations. Additional studies are needed to address cerebrovascular disease, life expectancy, and other problems related to Fabry disease. Enzyme replacement therapy with agalsidase beta has been a major advance in the care of patients with this disorder.

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Disclosure

C.E.P declares no competing financial interests. R.J.H. has done consulting work with Genzyme and Shire. He has been an investigator on clinical trials funded by Genzyme and Amicus. He serves on the medical advisory board for the Fabry Registry which is funded by Genzyme. He does not hold a financial interest in any company that produces treatments for Fabry disease or other lysosomal diseases, and does not receive salary support from any pharmaceutical company.

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Authors and Affiliations

  1. Division of Human Genetics, Cincinnati Children’s Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA

    Carlos E. Prada & Robert J. Hopkin

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  1. Carlos E. Prada
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  2. Robert J. Hopkin
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Correspondence to Robert J. Hopkin .

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Editors and Affiliations

  1. , Shaare Zedek Medical Center, Gaucher Clinic, Bayit Street 12, Jerusalem, 91034, Israel

    Deborah Elstein

  2. , Shaare Zedek Medical Center, Genetics Unit, Bayit Street 12, Jerusalem, 91031, Israel

    Gheona Altarescu

  3. University of Mainz, Children's Hospital, Langenbeckstrasse 1, Mainz, 55131, Germany

    Michael Beck

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Prada, C.E., Hopkin, R.J. (2010). Agalsidase Beta Clinical Trials and Long Term Experience. In: Elstein, D., Altarescu, G., Beck, M. (eds) Fabry Disease. Springer, Dordrecht. https://doi.org/10.1007/978-90-481-9033-1_26

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  • DOI: https://doi.org/10.1007/978-90-481-9033-1_26

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  • Publisher Name: Springer, Dordrecht

  • Print ISBN: 978-90-481-9032-4

  • Online ISBN: 978-90-481-9033-1

  • eBook Packages: MedicineMedicine (R0)

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