Long-term observational, non-randomized study of enzyme replacement therapy in late-onset glycogenos...
Long-term observational, non-randomized study of enzyme replacement therapy in late-onset glycogenosis type II
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Publisher
Dordrecht: Springer Netherlands
Journal title
Language
English
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Publisher
Dordrecht: Springer Netherlands
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Scope and Contents
Contents
Objectives
Type II glycogenosis (GSDII) is a lysosomal storage disorder due to acid alpha-glucosidase (GAA) deficiency. Enzyme replacement therapy (ERT) with human recombinant alpha-glucosidase (rhGAA) has been demonstrated to be effective in the treatment of infantile forms of GSDII, but little information is available concerning late-onset phe...
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Full title
Long-term observational, non-randomized study of enzyme replacement therapy in late-onset glycogenosis type II
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Record Identifier
TN_cdi_proquest_miscellaneous_815546433
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_815546433
Other Identifiers
ISSN
0141-8955,1573-2665
E-ISSN
1573-2665
DOI
10.1007/s10545-010-9201-8
