Lysosomal Diseases and Therapeutic Options: An Overview

  • Eveline J. Langereis
  • Frits A. WijburgEmail author
Part of the Stem Cell Biology and Regenerative Medicine book series (STEMCELL)


Lysosomal diseases cover a wide spectrum of inborn metabolic disorders and are characterized by a remarkable variability in pathophysiology and clinical presentation. This first chapter presents an overview of the natural history and biochemical characteristics of lysosomal storage disorders and some of the developments in therapy that have been made in the past decades and a brief look at potential future therapies.


Enzyme Replacement Therapy Lysosomal Enzyme Fabry Disease Gauche Disease Glycogen Storage Disease 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.


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Copyright information

© Springer Science+Business Media New York 2013

Authors and Affiliations

  1. 1.Department of PediatricsAcademic Medical CenterAmsterdamThe Netherlands

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