Safe and Successful Treatment With Agalsidase Beta During Pregnancy in Fabry Disease [PDF]
WOS: 000363832600010PubMed: 26338166Fabry disease, an X-linked lysosomal storage disorder, is caused by alpha-galactosidase A deficiency and leads to accumulation of glycospinhgolipids in most tissues, with life-theratening consequences in the kidney ...
Bahçebaşı, Zerrin Bicik +1 more
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Fabry\u27s disease is an X-linked inborn metabolic error caused by a deficiency of lysosomal ?- galactosidase A. Globotriaosylceramide (Gb3) accumulates in lysosomes of various cells.
R. Ebner; P. Rozenfeld; O. Croxatto; C. Fossati
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Outcomes from 5 years of treatment with agalsidase alfa enzyme replacement therapy (ERT) for Fabry disease in patients enrolled in the Fabry Outcome Survey (FOS) were compared with published findings for untreated patients with Fabry disease.
Beck, Michael +10 more
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Adequate hemodialysis does not compromise the cardioprotective effect of agalsidase alfa on patients with Fabry disease: a case report. [PDF]
Wei X, Jiang S, Zhuo L, Jiang H, Li W.
europepmc +1 more source
Altres ajuts: This work was supported by Shire International GmbH.This is a retrospective analysis of Fabry Outcome Survey data from children/adults (n = 677) receiving agalsidase alfa enzyme replacement therapy for a median of 3 years, examining ...
West, Michael L. +7 more
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Successful treatment using agalsidase alfa of a patient with Fabry disease who had anaphylaxis after agalsidase beta: A case report. [PDF]
Cakmak ME.
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Effect and Tolerability of Agalsidase Alfa in Patients with Fabry Disease Who Were Treatment Naïve or Formerly Treated with Agalsidase Beta or Agalsidase Alfa. [PDF]
Goker-Alpan O +7 more
europepmc +1 more source
Fabry cardiomyopathy presenting as hypertrophic phenotype with left ventricular outflow tract obstruction: a case series. [PDF]
Hosyanto FF, Li Y, Luo S, Huang B.
europepmc +1 more source
Erratum to: Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years' treatment. [PDF]
Kampmann C, Perrin A, Beck M.
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Impaired motor unit profile in Fabry disease: A quantitative electrophysiological study. [PDF]
Gunduz A +4 more
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