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Therapeutic goals in Fabry disease: Recommendations of a European expert panel, based on current clinical evidence with enzyme replacement therapy.

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a Division of Nephrology, University Clinic, University of Würzburg, Würzburg, Germany b French Referral Center for Fabry Disease, Division of Medical Genetics and INSERM U1179, University of Versailles, Paris-Saclay University,… Click to show full abstract

a Division of Nephrology, University Clinic, University of Würzburg, Würzburg, Germany b French Referral Center for Fabry Disease, Division of Medical Genetics and INSERM U1179, University of Versailles, Paris-Saclay University, Montigny, France c Department of Neurology, University of Erlangen-Nuremberg, Erlangen, Germany d Department of Paediatrics, University of Torino, Torino, Italy e INSERM U1018, University of Paris-Sud, University of Paris-Descartes, Paris, France University College London and Barts Heart Centre, London, United Kingdom

Keywords: goals fabry; genetics; fabry disease; therapeutic goals; university

Journal Title: Molecular genetics and metabolism
Year Published: 2019

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