A 15-Year Perspective of the Fabry Outcome Survey
AUTOR(ES)
Giugliani, Roberto
FONTE
J. inborn errors metab. screen.
DATA DE PUBLICAÇÃO
30/05/2019
RESUMO
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.
Documentos Relacionados
- Fascioliasis in buffaloes: A 5-year forecast analysis of the disease based on a 15-year survey in Brazil
- Clinicopathological analysis of salivary gland tumors over a 15-year period
- Salivary Gland Disorders in Children and Adolescents: A 15-year Experience
- Monthly variation in diagnosis of acute lymphoblastic leukemia and survival outcome in children and adults: 15-year trends at a single center
- Management of gastric remnant carcinoma based on the results of a 15-year endoscopic screening program.
