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BIOCHEMICAL AND PHARMACOLOGICAL CHARACTERIZATION OF DIFFERENT RECOMBINANT ACID α-GLUCOSIDASE PREPARATIONS EVALUATED FOR THE TREATMENT OF POMPE DISEASE
Pompe disease results in the accumulation of lysosomal glycogen in multiple tissues due to a deficiency of acid alpha-glucosidase (GAA). Enzyme replacement therapy for Pompe disease was recently approved in Europe, the U.S., Canada and Japan using a recombinant human GAA (Myozyme, alglucosidase alfa...
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| Main Authors: | , , , , , , , , , , , , , |
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| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
2008
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2774491/ https://ncbi.nlm.nih.gov/pubmed/18538603 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2008.04.009 |
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