Vol 79, No 12 (2021)
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 Dostálová1, Helena Hulkova23, Ales Linhart1
Pubmed: 34668177
Kardiol Pol 2021;79(12):1385-1386.

Abstract

Not available

References

  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.



Polish Heart Journal (Kardiologia Polska)