A 15-Year Perspective of the Fabry Outcome Survey
J. inborn errors metab. screen
;
4: e160041, 2016. tab, graf
Article
in English
|
LILACS-Express
| LILACS
| ID: biblio-1090901
ABSTRACT
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 (agalα). Established in 2001, FOS provides long-term data on agalα 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.
Full text:
Available
Index:
LILACS (Americas)
Type of study:
Prognostic study
/
Risk factors
Language:
English
Journal:
J. inborn errors metab. screen
Journal subject:
Medicina Cl¡nica
/
Patologia
Year:
2016
Type:
Article
Affiliation country:
Australia
/
Brazil
/
Canada
/
Germany
/
Spain
/
Switzerland
/
Taiwan
/
United kingdom
Institution/Affiliation country:
Dalhousie University/CA
/
Dep Genet UFRGS and INAGEMP/BR
/
National Yang-Ming University/TW
/
Shire/CH
/
Universitat Autònoma de Barcelona/ES
/
University College of London/GB
/
University of Mainz/DE
/
University of Melbourne/AU
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