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Substrate reduction therapy.

Abstract:

UNLABELLED: The therapeutic options for lysosomal storage diseases (LSDs) have expanded greatly over the past decade, although for many disorders there is still no effective treatment. Given that the majority of LSDs involve pathological changes in both the brain and peripheral tissues, effective treatment of central nervous system (CNS) and peripheral manifestations still remains a considerable technical challenge. Type 1 Gaucher disease has two approved treatment modalities - enzyme replac...

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Publication status:
Published

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Institution:
University of Oxford
Division:
MSD
Department:
Pharmacology
Role:
Author
Volume:
97
Issue:
457
Pages:
88-93
Host title:
Acta paediatrica (Oslo, Norway : 1992). Supplement
Publication date:
2008-04-01
Event location:
Norway
DOI:
EISSN:
1651-2227
ISSN:
0803-5326
Source identifiers:
185124
Keywords:
Pubs id:
pubs:185124
UUID:
uuid:ca8b3d7b-dfc3-4975-86dc-82864e010331
Local pid:
pubs:185124
Deposit date:
2012-12-19

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