Twenty years of the Fabry Outcome Survey (FOS): insights, achievements, and lessons learned from a global patient registry

Twenty years of the Fabry Outcome Survey (FOS): insights, achievements, and lessons learned from a global patient registry

Abstract Background Patient registries provide long-term, real-world evidence that aids the understanding of the natural history and progression of disease, and the effects of treatment on large patient populations with rare diseases. The year 2021 marks the 20th anniversary of the Fabry Outcome Sur...

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Bibliographic Details
Main Authors: Michael Beck, Uma Ramaswami, Elizabeth Hernberg-Ståhl, Derralynn A. Hughes, Christoph Kampmann, Atul B. Mehta, Kathleen Nicholls, Dau-Ming Niu, Guillem Pintos-Morell, Ricardo Reisin, Michael L. West, Jörn Schenk, Christina Anagnostopoulou, Jaco Botha, Roberto Giugliani
Format: Article
Language:English
Published: BMC 2022-06-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Agalsidase alfa
Enzyme replacement therapy
Fabry disease
Cardiovascular outcomes
Renal outcomes
Online Access:https://doi.org/10.1186/s13023-022-02392-9

Internet

https://doi.org/10.1186/s13023-022-02392-9

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