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Enzyme Replacement or Substrate Reduction? A Review of Gaucher Disease Treatment Options
Background: Gaucher disease is a rare lysosomal storage disease resulting from a deficiency or reduced activity in the acid β-glucocosidase enzyme. Only 1 treatment option was available for 15 years, but several new treatment options have come to market since 2003. Objective: The article will detail...
Sparad:
| I publikationen: | Hosp Pharm |
|---|---|
| Huvudupphovsmän: | , |
| Materialtyp: | Artigo |
| Språk: | Inglês |
| Publicerad: |
Thomas Land Publishers, Inc.
2016
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| Ämnen: | |
| Länkar: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4981103/ https://ncbi.nlm.nih.gov/pubmed/27559188 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1310/hpj5107-553 |
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