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Clinical vignette
Published online: 2021-10-15
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Anderson–Fabry disease — no histological signs of pathological accumulation in arterial and venous endothelium during pegunigalsidase alfa therapy

Gabriela Dostalova1, Helena Hulkova23, Ales Linhart1
DOI: 10.33963/KP.a2021.0139
·
Pubmed: 34668177
Affiliations
  1. 2nd Department of Medicine — Department of Cardiovascular Medicine, 1st Faculty of Medicine, Charles University in Prague and General University Hospital in Prague, Czech Republic
  2. Department of Pediatrics and Adolescent Medicine, 1st Faculty of Medicine, Charles University in Prague and General University Hospital in Prague, Czech Republic
  3. Department of Pathology, 1st Faculty of Medicine, Charles University in Prague and General University Hospital in Prague, Czech Republic

open access

Online first
Clinical vignette
Published online: 2021-10-15

Abstract

Not available

Abstract

Not available
Get Citation
About this article
Title

Anderson–Fabry disease — no histological signs of pathological accumulation in arterial and venous endothelium during pegunigalsidase alfa therapy

Journal

Kardiologia Polska (Polish Heart Journal)

Issue

Online first

Article type

Clinical vignette

Published online

2021-10-15

DOI

10.33963/KP.a2021.0139

Pubmed

34668177

Authors

Gabriela Dostalova
Helena Hulkova
Ales Linhart

References (5)
  1. Saito S, Ohno K, Sakuraba H. Fabry-database.org: database of the clinical phenotypes, genotypes and mutant α-galactosidase A structures in Fabry disease. J Hum Genet. 2011; 56(6): 467–468.
  2. Elleder M. Sequelae of storage in Fabry disease — pathology and comparison with other lysosomal storage diseases. Acta Paediatr Suppl. 2003; 92(443): 46–53; discussion 45.
  3. Nowicki M, Bazan-Socha S, Błażejewska-Hyzorek B, et al. Enzyme replacement therapy in Fabry disease in Poland: a position statement. Pol Arch Intern Med. 2020; 130(1): 91–97.
  4. Linhart A, Dostalova G, Nicholls K, et al. Switching from agalsidase alfa to pegunigalsidase alfa for treating Fabry disease: one year of treatment data from BRIDGE, a phase III open label study. Mol Genet Metab. 2020; 129(2): S98–S99.
  5. Schiffmann R, Goker-Alpan O, Holida M, et al. Pegunigalsidase alfa, a novel pegylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: a 1-year phase 1/2 clinical trial. J Inherit Metab Dis. 2019; 42(3): 534–544.

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