Giugliani, R;
Niu, DM;
Ramaswami, U;
West, M;
Hughes, D;
Kampmann, C;
Pintos-Morell, G;
... Beck, M; + view all
(2016)
A 15-year perspective of the fabry outcome survey.
Journal of Inborn Errors of Metabolism and Screening
, 4
10.1177/2326409816666298.
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Abstract
The Fabry Outcome Survey (FOS) is an international long-term observational registry sponsored by Shire for patients diagnosed with Fabry disease who are receiving or are candidates for therapy with agalsidase alfa (agala). Established in 2001, FOS provides long-term data on agala safety/efficacy and collects data on the natural history of Fabry disease, with the aim of improving clinical management. The FOS publications have helped establish prognostic and severity scores, defined the incidence of specific disease variants and implications for clinical management, described clinical manifestations in special populations, confirmed the high prevalence of cardiac morbidity, and demonstrated correlations between ocular changes and Fabry disease severity. These FOS data represent a rich resource with utility not only for description of natural history/therapeutic effects but also for exploratory hypothesis testing and generation of tools for diagnosis/management, with the potential to improve future patient outcomes.
| Type: | Article |
|---|---|
| Title: | A 15-year perspective of the fabry outcome survey |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1177/2326409816666298 |
| Publisher version: | https://doi.org/10.1177/2326409816666298 |
| Language: | English |
| Additional information: | This article is distributed under the terms of the Creative Commons Attribution 3.0 License (http://www.creativecommons.org/licenses/by/3.0/) which permits any use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access pages (https://us.sagepub.com/en-us/nam/open-access-at-sage). |
| Keywords: | agalsidase alfa, enzyme replacement therapy, Fabry disease, Fabry Outcome Survey, outcomes |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > Research Department of Haematology |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10045911 |
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