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Predicting Cross Reactive Immunological Material (CRIM) Status in Pompe Disease Using GAA Mutations: Lessons Learned from 10 Years of Clinical Laboratory Testing Experience
Enzyme replacement therapy (ERT) for Pompe disease using recombinant acid alpha-glucosidase (rhGAA) has resulted in increased survival although the clinical response is variable. Cross Reactive Immunological Material (CRIM)-negative status has been recognized as a poor prognostic factor. CRIM-negati...
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| Hoofdauteurs: | , , , , , , |
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| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
2012
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| Onderwerpen: | |
| Online toegang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3278076/ https://ncbi.nlm.nih.gov/pubmed/22252923 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ajmg.c.31319 |
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