Lysosomal Storage Disorders (ePub)

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.

The first part of the book reviews and classifies our current
understanding of the physiology and pathophysiology of lysosomal
storage disorders. The second part of the book reviews individual
diseases, and gives perspectives from patients and experts looking
towards future therapeutic directions.

Lysosomal Storage Disorders: A Practical Guide is the
ideal guide for a wide audience including scientists, clinicians,
health care workers and administrators, those working in the
pharmaceutical industry, patients and their organisations.

Titles of related interest

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Atlas of Endocrine and Metabolic Disease * Pozzilli * ISBN 9780470656273