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Enzyme replacement therapy

Mannose 6-Phosphate Receptor and Sortilin Mediated Endocytosis of α-Galactosidase A in Kidney Endothelial Cells

Thaneas Prabakaran, Rikke Nielsen, [ ... ], Erik I. Christensen

Cardiomyopathy and Response to Enzyme Replacement Therapy in a Male Mouse Model for Fabry Disease

Aurelie Nguyen Dinh Cat, Brigitte Escoubet, [ ... ], Frederic Jaisser

Globotriaosylsphingosine Accumulation and Not Alpha-Galactosidase-A Deficiency Causes Endothelial Dysfunction in Fabry Disease

Mehdi Namdar, Catherine Gebhard, [ ... ], Giovanni G. Camici

Anti-TNF-Alpha Therapy Enhances the Effects of Enzyme Replacement Therapy in Rats with Mucopolysaccharidosis Type VI

Efrat Eliyahu, Theodore Wolfson, [ ... ], Calogera M. Simonaro

A Distinct Urinary Biomarker Pattern Characteristic of Female Fabry Patients That Mirrors Response to Enzyme Replacement Therapy

Andreas D. Kistler, Justyna Siwy, [ ... ], Andreas L. Serra

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