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Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?
Lysosomal storage diseases (LSDs), as a group, are among the most common inherited diseases affecting children. The primary defect is typically a genetic deficiency of one of the lysosomal enzymes, often causing accumulation of undegraded substrates within the lysosome. This accumulation causes nume...
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| Hoofdauteurs: | , , |
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| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
Oxford University Press
2011
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| Onderwerpen: | |
| Online toegang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3095053/ https://ncbi.nlm.nih.gov/pubmed/21421999 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddr112 |
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