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Fabry Disease

Perspectives from 5 Years of FOS

Editors: Atul Mehta, Michael Beck, and Gere Sunder-Plassmann.

Editor Information
Oxford: Oxford PharmaGenesis; .
ISBN-10: 1-903539-03-X

Published to commemorate the first 5 years of the Fabry Outome Survey, this volume brings together contributions from leading experts in the field of lysosomal storage diseases (LSDs) in general and Fabry disease in particular in a single state-of-the-art publication. The first section covers general aspects of LSDs, with chapters on topics as diverse as the cellular pathophysiology of lysosomes, the development of enzyme replacement therapy (ERT), the central role played by patient groups, and the regulatory framework governing the treatment of orphan diseases. The second section describes the role of observational outcome surveys and the organization and development of FOS. The third and fourth sections draw largely, but not exclusively, from data in FOS. They describe the clinical features and natural course of Fabry disease and the multiple beneficial effects of ERT with agalsidase alfa on the function of affected organs and quality of life.

By the end of 2005, FOS contained comprehensive information on over 750 patients from 13 countries. This outcome survey has therefore been able to greatly extend the information previously available from limited small-scale clinical trials, and reflects the clinical picture of Fabry disease and its response to ERT with agalsidase alfa within the context of normal clinical practice.


Supported by an unrestricted educational grant by Shire HGT.

The information contained in this publication does not necessarily reflect the opinions or recommendations of the publishers or sponsors. The dosages, indications and methods of use for the product(s) referred to by the authors may not necessarily be those indicated on the Summary of Product Characteristics or the US Prescribing Information for the product(s) and may reflect the clinical experience of the authors or may be derived from the pharmaceutical literature or other clinical sources. When diagnosing and treating patients, doctors should take into account the individual patient's condition(s) and should consult officially approved monographs such as the Summary of Product Characteristics or US Prescribing Information prior to following any procedures or treatments based on data presented in this book.

Copyright © 2006, Oxford PharmaGenesis™.
Bookshelf ID: NBK11586PMID: 21290683


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