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Review

Novel therapeutic targets for the treatment of Fabry disease

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Pages 821-833 | Published online: 15 May 2007

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BE Smid, JMFG Aerts, RG Boot, GE Linthorst & CEM Hollak. (2010) Pharmacological small molecules for the treatment of lysosomal storage disorders. Expert Opinion on Investigational Drugs 19:11, pages 1367-1379.
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Articles from other publishers (11)

Albina Nowak, Olivier Dormond, Véronique Monzambani, Uyen Huynh-Do & Frédéric Barbey. (2022) Agalsidase-β should be proposed as first line therapy in classic male Fabry patients with undetectable α-galactosidase A activity. Molecular Genetics and Metabolism 137:1-2, pages 173-178.
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Doris Klingelhöfer, Markus Braun, Rebekka K. Seeger‐Zybok, David Quarcoo, Dörthe Brüggmann & David A. Groneberg. (2020) Global research on Fabry's disease: Demands for a rare disease. Molecular Genetics & Genomic Medicine 8:9.
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Marie-Laure Boof, Atef Halabi, Mike Ufer & Jasper Dingemanse. (2019) Impact of the organic cation transporter 2 inhibitor cimetidine on the single-dose pharmacokinetics of the glucosylceramide synthase inhibitor lucerastat in healthy subjects. European Journal of Clinical Pharmacology 76:3, pages 431-437.
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Maria J. Ferraz, Wouter W. Kallemeijn, Mina Mirzaian, Daniela Herrera Moro, Andre Marques, Patrick Wisse, Rolf G. Boot, Lianne I. Willems, H.S. Overkleeft & J.M. Aerts. (2014) Gaucher disease and Fabry disease: New markers and insights in pathophysiology for two distinct glycosphingolipidoses. Biochimica et Biophysica Acta (BBA) - Molecular and Cell Biology of Lipids 1841:5, pages 811-825.
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Thomas E. Lloyd & Vinay Chaudhry. 2011. Neuromuscular Disorders: Treatment and Management. Neuromuscular Disorders: Treatment and Management 191 213 .
Cintia L. Marchesoni, Norma Roa, Ana Mar?a Pardal, Pablo Neumann, Guillermo C?ceres, Pablo Mart?nez, Isaac Kisinovsky, Silvia Bianchi, Ana L?a Tarabuso & Ricardo C. Reisin. (2010) Misdiagnosis in Fabry Disease. The Journal of Pediatrics 156:5, pages 828-831.
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Ana Maria Martins, V?nia D'Almeida, Sandra Obikawa Kyosen, Edna Tiemi Takata, Alvimar Gon?alves Delgado, ?ngela Maria Barbosa Ferreira Gon?alves, Caio C?sar Benetti Filho, Dino Martini Filho, Gilson Biagini, Helena Pimentel, Hugo Abensur, Humberto Cenci Guimar?es, Jaelson Guilhem Gomes, Jos? Sobral Neto, Luiz Oct?vio Dias D'Almeida, Luiz Roberto Carvalho, Maria Beatriz Harouche, Maria Cristina Jacometti Maldonado, Osvaldo J.M. Nascimento, Paulo Sergio dos Santos Montoril & Ricardo Villela Bastos. (2009) Guidelines to Diagnosis and Monitoring of Fabry Disease and Review of Treatment Experiences. The Journal of Pediatrics 155:4, pages S19-S31.
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Orna Staretz-Chacham, Tess C. Lang, Mary E. LaMarca, Donna Krasnewich & Ellen Sidransky. (2009) Lysosomal Storage Disorders in the Newborn. Pediatrics 123:4, pages 1191-1207.
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Ulrich Matzner, Renate L?llmann-Rauch, Stijn Stroobants, Claes Andersson, Cecilia Weigelt, Carl Eistrup, Jens Fogh, Rudi D'Hooge & Volkmar Gieselmann. (2009) Enzyme Replacement Improves Ataxic Gait and Central Nervous System Histopathology in a Mouse Model of Metachromatic Leukodystrophy. Molecular Therapy 17:4, pages 600-606.
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Jana Keslová-Veselíková, Helena Hůlková, Robert Dobrovolný, Befekadu Asfaw, Helena Poupětová, Linda Berná, Jakub Sikora, Lubor Goláň, Jana Ledvinová & Milan Elleder. (2008) Replacement of α-galactosidase A in Fabry disease: effect on fibroblast cultures compared with biopsied tissues of treated patients. Virchows Archiv 452:6, pages 651-665.
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Johannes M Aerts, Marielle J van Breemen, Anton P Bussink, Karen Ghauharali, Richard Sprenger, Rolf G Boot, Johanna E Groener, Carla E Hollak, Mario Maas, Suzanne Smit, Huub C Hoefsloot, Age K Smilde, Johannes PC Vissers, Sheryas de Jong, Dave Speijer & Chris G de Koster. (2008) Biomarkers for lysosomal storage disorders: identification and application as exemplified by chitotriosidase in Gaucher disease. Acta Paediatrica 97:s457, pages 7-14.
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