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Fabry Disease: Molecular Basis, Pathophysiology, Diagnostics and Potential Therapeutic Directions2021 Biomolecules article Medicine Lysosomal Storage Disorders Research Open access cited by 1 patent
Ken Kok, Kimberley C. Zwiers, Rolf G. Boot, Herman S. Overkleeft, Johannes M. F. G. Aerts, Marta Artola
131citations -
Preclinical efficacy and safety of adeno-associated virus 5 alpha-galactosidase: A gene therapy for Fabry disease2024 Molecular Therapy — Methods & Clinical Development article Medicine Lysosomal Storage Disorders Research Open access
Jolanda M.P. Liefhebber, Giso Brasser, Elisabeth A. Spronck, Roelof Ottenhoff, Lieke Paerels, Maria J. Ferraz, +6 more
9citations -
Unraveling A4GALT Mechanism and Its Modulation With Adamantyl‐Galactosylceramide Analogues: Advancing Fabry Disease Therapeutic Strategies2026 Angewandte Chemie International Edition article Medicine Lysosomal Storage Disorders Research Open access
Nicky de Koster, Òscar Vidal-Gironès, Rowan de Graaf, Ken Kok, Lindsey T. Lelieveld, Jackelien Hoogervorst, +8 more
1citations -
Therapeutic challenges in two adolescent male patients with Fabry disease and high antibody titres2020 Molecular Genetics and Metabolism Reports article Medicine Lysosomal Storage Disorders Research Open access
Aizeddin A. Mhanni, Christiane Auray‐Blais, Michel Boutin, Alie J. Johnston, Kaye M. LeMoine, Johannes M. F. G. Aerts, +2 more
8citations -
Right ventricular strain in Fabry disease: Prognostic implications2022 International Journal of Cardiology article Medicine Lysosomal Storage Disorders Research Open access
Maria Chiara Meucci, Rosa Lillo, Federica Mango, Antonella Lombardo, Gaetano Antonio Lanza, Valentina Parisi, +5 more
6citations -
Comparative analysis of right ventricular strain in Fabry cardiomyopathy and sarcomeric hypertrophic cardiomyopathy2022 European Heart Journal - Cardiovascular Imaging article Medicine Cardiomyopathy and Myosin Studies Open access
Maria Chiara Meucci, Rosa Lillo, Antonella Lombardo, Gaetano Antonio Lanza, Marianne Bootsma, Steele C. Butcher, +6 more
22citations -
Monogenic cerebral small‐vessel diseases: diagnosis and therapy. Consensus recommendations of the European Academy of Neurology2020 European Journal of Neurology article Medicine Cerebrovascular and genetic disorders Open access
Michelangelo Mancuso, Marcel Arnold, Anna Bersano, Alessandro P. Burlina, Hugues Chabriat, Stéphanie Debette, +8 more
194citations -
Lyso−Gb is not a predictive biomarker of treatment response in migalastat-treated patients with migalastat-amenable variants2020 Molecular Genetics and Metabolism article Medicine Lysosomal Storage Disorders Research Open access
Raphael Schiffmann, Daniel Georges Bichet, Johannes M. F. G. Aerts, Nina Skuban, Atul B Mehta
0citations