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Mehta A, Beck M, Sunder-Plassmann G, editors. Fabry Disease: Perspectives from 5 Years of FOS. Oxford: Oxford PharmaGenesis; 2006.
1. History of lysosomal storage diseases: an overview
2. Epidemiology of lysosomal storage diseases: an overview
4. Cellular pathophysiology of lysosomal storage diseases
5. Importance of glycosylation in enzyme replacement therapy
6. Animal models of lysosomal storage diseases: their development and clinical relevance
7. General aspects of X-linked diseases
8. Laboratory diagnosis of lysosomal storage diseases
9. Biomarkers in lysosomal storage diseases
10. Enzyme replacement therapy – a brief history
11. Regulatory framework for the treatment of orphan diseases
12. Role of patient support groups in lysosomal storage diseases
- General aspects of lysosomal storage diseases - Fabry DiseaseGeneral aspects of lysosomal storage diseases - Fabry Disease
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