- 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...
Expand abstract - Publication status:
- Published
- Publisher copy:
- 10.1111/j.1651-2227.2008.00656.x
-
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- Volume:
- 97
- Issue:
- 457
- Pages:
- 88-93
- Publication date:
- 2008-04-05
- DOI:
- EISSN:
-
1651-2227
- ISSN:
-
0803-5326
- URN:
-
uuid:ca8b3d7b-dfc3-4975-86dc-82864e010331
- Source identifiers:
-
185124
- Local pid:
- pubs:185124
- Copyright date:
- 2008
Conference
Substrate reduction therapy.
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