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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...
Gardado en:
| Publicado en: | J Inherit Metab Dis |
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| Main Authors: | , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado: |
Springer Netherlands
2014
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| Assuntos: | |
| Acceso en liña: | 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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