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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Bibliographic Details
Main Authors: Porto, Caterina, Cardone, Monica, Fontana, Federica, Rossi, Barbara, Tuzzi, Maria Rosaria, Tarallo, Antonietta, Barone, Maria Vittoria, Andria, Generoso, Parenti, Giancarlo
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2009
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Original Articles
Online Access: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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