Conference item
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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- Publisher copy:
- 10.1111/j.1651-2227.2008.00656.x
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Bibliographic Details
- 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
Item Description
- 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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- Copyright date:
- 2008
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