Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease

BACKGROUND: Enzyme-replacement therapy (ERT) in Pompe disease—an inherited metabolic disorder caused by acid α-glucosidase deficiency and characterized in infants by generalized muscle weakness and cardiomyopathy—can be complicated by immune responses. Infants that do not produce any endogenous acid...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:J Inherit Metab Dis
Egile Nagusiak: van Gelder, Carin M., Hoogeveen-Westerveld, Marianne, Kroos, Marian A., Plug, Iris, van der Ploeg, Ans T., Reuser, Arnold J. J.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: Springer Netherlands 2014
Gaiak:
Original Article
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC4341007/
https://ncbi.nlm.nih.gov/pubmed/24715333
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-014-9707-6
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