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Drug Design, Development and Therapy Volume 13, 2019 - Issue
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Original Research

Cardio- Renal Outcomes With Long- Term Agalsidase Alfa Enzyme Replacement Therapy: A 10- Year Fabry Outcome Survey (FOS) Analysis

Uma Ramaswami1 Royal Free London NHS Foundation Trust, Lysosomal Disorders Unit, Institute of Immunity and Transplantation, London, UKCorrespondence[email protected]
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Michael Beck2 Centre for Paediatric and Adolescent Medicine, University Medical Centre, University of Mainz, Mainz, GermanyView further author information
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Derralynn Hughes1 Royal Free London NHS Foundation Trust, Lysosomal Disorders Unit, Institute of Immunity and Transplantation, London, UKView further author information
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Christoph Kampmann2 Centre for Paediatric and Adolescent Medicine, University Medical Centre, University of Mainz, Mainz, GermanyView further author information
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Jaco Botha3 Department of Biostatistics and Programming, Takeda, Zug, SwitzerlandView further author information
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Guillem Pintos-Morell4 Centre for Rare Diseases, University Hospital Vall d’Hebron, Barcelona, CIBERER-GCV08_IGTP Research Institute and Teaching Unit Germans Trias i Pujol, Universitat Autònoma de Barcelona, Badalona, SpainORCID Iconhttps://orcid.org/0000-0002-9347-2386View further author information
ORCID Icon,
Michael L West5 Department of Medicine, Dalhousie University, Halifax, Nova Scotia, CanadaORCID Iconhttps://orcid.org/0000-0003-4897-1378View further author information
ORCID Icon,
Dau-Ming Niu6 Department of Paediatrics, Taipei Veterans General Hospital, Taipei, TaiwanView further author information
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Kathy Nicholls7 Department of Nephrology, Royal Melbourne Hospital, University of Melbourne, Parkville, VIC, Australia;8 Department of Medicine, Royal Melbourne Hospital, University of Melbourne, Parkville, VIC, AustraliaORCID Iconhttps://orcid.org/0000-0002-1297-8722View further author information
ORCID Icon,
Roberto Giugliani9 Medical Genetics Service HCPA, Department of Genetics, UFRGS, and INAGEMP, Porto Alegre, BrazilORCID Iconhttps://orcid.org/0000-0001-9655-3686View further author information
ORCID Icon & On behalf of the FOS Study Group show all
Pages 3705-3715 | Published online: 25 Oct 2019

References

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  • Germain DP, Charrow J, Desnick RJ, et al. Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease. J Med Genet. 2015;52(5):353–358. doi:10.1136/jmedgenet-2014-10279725795794
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