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Gaucher disease: clinical profile and therapeutic developments
Gaucher disease is a rare inborn error of glycosphingolipid metabolism due to deficiency of lysosomal acid β-glucocerebrosidase; the condition has totemic significance for the development of orphan drugs. A designer therapy, which harnesses the mannose receptor to complement the functional defect in...
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| Formato: | Texto |
| Lenguaje: | English |
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Dove Medical Press
2010
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3010821/ https://www.ncbi.nlm.nih.gov/pubmed/21209725 http://dx.doi.org/10.2147/BTT.S7582 |