Global reach of over 20 years of experience in the patient-centered Fabry Registry: Advancement of Fabry disease expertise and dissemination of real-world evidence to the Fabry community-Reference-Cited by-同舟云学术

Global reach of over 20 years of experience in the patient-centered Fabry Registry: Advancement of Fabry disease expertise and dissemination of real-world evidence to the Fabry community

Published:2023-07 Issue:3 Volume:139 Page:107603
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Wanner Christoph,Ortiz Alberto,Wilcox William R.,Hopkin Robert J.,Johnson Jack,Ponce Elvira,Ebels Johan T.,Batista Julie L.,Maski Manish,Politei Juan M.,Martins Ana Maria,Banikazemi Maryam,Linhart Aleš,Mauer Michael,Oliveira João P.,Weidemann Frank,Germain Dominique P.

Funder

Sanofi-Aventis Deutschland

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

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4. Fabry’s disease: classification as a sphingolipidosis and partial characterization of a novel glycolipid;Sweeley;J. Biol. Chem.,1963

5. Enzymatic defect in Fabry's disease. Ceramidetrihexosidase deficiency;Brady;N. Engl. J. Med.,1967

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1. Case report: First diagnosis of Fabry disease in North Macedonia in a patient presenting with kidney failure on hemodialysis;Frontiers in Genetics;2024-08-14

2. Clinical outcomes in patients switching from agalsidase beta to migalastat: A Fabry Registry analysis;Journal of Inherited Metabolic Disease;2024-07-04

3. Reduction in kidney function decline and risk of severe clinical events in agalsidase beta–treated Fabry disease patients: a matched analysis from the Fabry Registry;Clinical Kidney Journal;2024-07-02

4. The Italian Fabry Disease Cardiovascular Registry (IFDCR);European Heart Journal - Quality of Care and Clinical Outcomes;2024-06-27

5. Pegunigalsidase alfa: a novel, pegylated recombinant alpha-galactosidase enzyme for the treatment of Fabry disease;Frontiers in Genetics;2024-04-12