The Pharmacological Chaperone N-butyldeoxynojirimycin Enhances Enzyme Replacement Therapy in Pompe Disease Fibroblasts

In spite of the progress in the treatment of lysosomal storage diseases (LSDs), in some of these disorders the available therapies show limited efficacy and a need exists to identify novel therapeutic strategies. We studied the combination of enzyme replacement and enzyme enhancement by pharmacologi...

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Main Authors: Porto, Caterina, Cardone, Monica, Fontana, Federica, Rossi, Barbara, Tuzzi, Maria Rosaria, Tarallo, Antonietta, Barone, Maria Vittoria, Andria, Generoso, Parenti, Giancarlo
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2009
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Original Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2835191/
https://ncbi.nlm.nih.gov/pubmed/19293774
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.53
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