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Cardio-renal outcomes with long-term agalsidase alfa enzyme replacement therapy : A 10-year fabry outcome survey (FOS) analysis / Ramaswami, Uma (Royal Free London NHS Foundation Trust. Lysosomal Disorders Unit. Institute of Immunity and Transplantation) ; Beck, Michael (Centre for Paediatric and Adolescent Medicine. University Medical Centre. University of Mainz) ; Hughes, D. (Royal Free London NHS Foundation Trust. Lysosomal Disorders Unit. Institute of Immunity and Transplantation) ; Kampmann, C. (Centre for Paediatric and Adolescent Medicine. University Medical Centre. University of Mainz) ; Botha, J. (Department of Biostatistics and Programming. Takeda) ; Pintos-Morell, Guillem (Institut Germans Trias i Pujol. Hospital Universitari Germans Trias i Pujol) ; West, M. L. (Department of Medicine. Dalhousie University) ; Niu, D. M. (Department of Paediatrics. Taipei Veterans General Hospital) ; Nicholls, K. (Royal Melbourne Hospital (Melbourne, Austràlia)) ; Giugliani, R. (Medical Genetics Service HCPA. Department of Genetics. UFRGS. INAGEMP) ; Universitat Autònoma de Barcelona
Purpose: Following the publication of 5-year agalsidase alfa enzyme replacement therapy (ERT) outcomes data from the Fabry Outcome Survey (FOS), 10-year data were analyzed. Patients and methods: FOS (ClinicalTrials. [...]
2019 - DOI (Digital Object Identifier)

10.2147/DDDT.S207856
Drug Design, Development and Therapy, Vol. 13 (2019) , p. 3705-3715

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