Lysosomal storage disorders : a practical guide /
Awareness of lysomal storage disorders needs to be raised and there is very substantial pharmaceutical interest to do so. The disorders are often viewed as obscurities but in fact they are treatable. Enzyme replacement therapy is available for four of the disorders and will be available for a furthe...
| Other Authors: | , |
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| Format: | eBook |
| Language: | English |
| Published: |
Chichester, West Sussex :
Wiley-Blackwell,
2013.
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| Subjects: | |
| Online Access: | Full Text via HEAL-Link |
| Summary: | Awareness of lysomal storage disorders needs to be raised and there is very substantial pharmaceutical interest to do so. The disorders are often viewed as obscurities but in fact they are treatable. Enzyme replacement therapy is available for four of the disorders and will be available for a further three disorders in the course of the next year. Substrate reduction therapy is licensed for one of them but in the course of the next 12 months it will be licensed for two others and a new form of substrate reduction therapy is being introduced. These diseases present to a very wide range of phy. |
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| Physical Description: | 1 online resource |
| Bibliography: | Includes bibliographical references and index. |
| ISBN: | 9781118514641 1118514645 9781118514672 111851467X 9781283644419 128364441X 9781118514696 1118514696 |
| DOI: | 10.1002/9781118514672 |
