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10 p, 2.0 MB Analysis of Renal and Cardiac Outcomes in Male Participants in the Fabry Outcome Survey Starting Agalsidase Alfa Enzyme Replacement Therapy Before and After 18 Years of Age / Parini, Rossella (Istituto di Ricovero e Cura A Carattere Scientifico (IRCCS)) ; Pintos-Morell, Guillem (Hospital Universitari Vall d'Hebron. Institut de Recerca) ; Hennermann, Julia B. (University Medical Center) ; Hsu, Ting-Rong (Taipei Veterans General Hospital) ; Karabul, Nesrin (University Children's Hospital and Centre for Rare Diseases) ; Kalampoki, Vasiliki (Shire (Suïssa)) ; Gurevich, Andrey (Shire (Suïssa)) ; Ramaswami, Uma (Royal Free London NHS Foundation Trust) ; Universitat Autònoma de Barcelona
To determine the impact of initiating enzyme replacement therapy (ERT) with agalsidase alfa early in the course of Fabry disease, we evaluated renal and cardiac outcomes for ≤10 years after ERT initiation in males from the Fabry Outcome Survey (FOS). [...]
2020 - 10.2147/DDDT.S249433
Drug Design, Development and Therapy, Vol. 14 (june 2020) , p. 2149-2158  

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