Mucopolysaccharidoses - A Guide for Physicians and Parents

In the past decade there has been enormous progress in genetics, biochemical principles and therapeutic options regarding mucopolysaccharidosis. The genetic bases of the diseases have been identified, the production of sufficient amounts of enzymes and enzyme replacement therapy have become possible. This book attempts to give an outline of biological and medical principles of mucopolysaccharidoses and also points out the most recent therapeutic alternatives. It is based on long-term experiences of the authors in this field who have been following their patients on their difficult journey for years. This guide informs in a comprehensive and intelligible way. Doctors of all departments ought to use it when treating children and adults with mucopolysaccharidoses and furthermore it is a helpful companion for patients and parents.