Two decades of experience of the Fabry Outcome Survey provides further confirmation of the long-term effectiveness of agalsidase alfa enzyme replacement therapy

Two decades of experience of the Fabry Outcome Survey provides further confirmation of the long-term effectiveness of agalsidase alfa enzyme replacement therapy

Background: Analyses of up to 20 years of data from the Fabry Outcome Survey (FOS) assessed the long-term effectiveness of agalsidase alfa enzyme replacement therapy. Methods: The impact of agalsidase alfa treatment on renal, cardiac, morbidity, and mortality outcomes in FOS was compared with untrea...

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Main Authors: Uma Ramaswami, Guillem Pintos-Morell, Christoph Kampmann, Kathleen Nicholls, Dau-Ming Niu, Ricardo Reisin, Michael L. West, Christina Anagnostopoulou, Jaco Botha, Dalia Jazukeviciene, Jörn Schenk, Derralynn A. Hughes, Roberto Giugliani
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Registry data
Clinical outcomes
Agalsidase alfa
Fabry disease
Long-term data
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426925000308
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