Immune responses to alglucosidase in infantile Pompe disease: recommendations from an Italian pediat...
Immune responses to alglucosidase in infantile Pompe disease: recommendations from an Italian pediatric expert panel
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Publisher
England: BioMed Central
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Language
English
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Publisher
England: BioMed Central
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Contents
Classic infantile onset of Pompe disease (c-IOPD) leads to hypotonia and hypertrophic cardiomyopathy within the first days to weeks of life and, without treatment, patients die of cardiorespiratory failure in their first 1-2 years of life. Enzymatic replacement therapy (ERT) with alglucosidase alfa is the only available treatment, but adverse immun...
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Full title
Immune responses to alglucosidase in infantile Pompe disease: recommendations from an Italian pediatric expert panel
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TN_cdi_doaj_primary_oai_doaj_org_article_61af1ccf535f4399a2639a84afdb50e6
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_61af1ccf535f4399a2639a84afdb50e6
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ISSN
1824-7288
E-ISSN
1824-7288
DOI
10.1186/s13052-022-01219-4
