Lysosomal Storage Disorders - A Practical Guide

Lysosomal Storage Disorders: A Practical Guide

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English | 208 pages | Wiley-Blackwell; 1st edition (January 22, 2013) | 0470670878 | PDF | 4.61 Mb

The last two decades have seen a huge expansion in research in the area of lysosomal storage disorders, which has substantially extended our understanding of both the scientific and the clinical basis of these diseases. Lysosomal Storage Disorders: A Practical Guide is the fruit of an ambitious project aiming to review both the scientific and the clinical aspects of lysosomal storage disorders, resulting in
this accessible volume, which gives an up-to-date overview of the subject.

There is substantial scientific interest in these diseases: new advances in small molecule therapy are likely to be useful in the near future, and trials are already underway. Lysosomal storage disorders offer a unique platform for teaching modern clinical science, from basic genetics through to clinical applications.