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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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Detalles Bibliográficos
Autor Principal: Cox, Timothy M
Formato: Artigo
Idioma:Inglês
Publicado: Dove Medical Press 2010
Assuntos:
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC3010821/
https://ncbi.nlm.nih.gov/pubmed/21209725
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/BTT.S7582
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