| Autor: |
Ankit K. Desai, Zoheb B. Kazi, Deeksha S. Bali, Priya S. Kishnani |
| Jazyk: |
angličtina |
| Rok vydání: |
2019 |
| Předmět: |
|
| Zdroj: |
Molecular Genetics and Metabolism Reports, Vol 20, Iss , Pp - (2019) |
| Druh dokumentu: |
article |
| ISSN: |
2214-4269 |
| DOI: |
10.1016/j.ymgmr.2019.100475 |
| Popis: |
Enzyme replacement therapy (ERT) with rhGAA has improved clinical outcomes in infantile Pompe disease (IPD). A subset of CRIM-positive IPD patients develop high and sustained antibody titers (HSAT; ≥51,200) and/or sustained intermediate titer (SIT; ≥12,800 and 6 months of antibody titer data available, were included in the study. Patients were classified based on their longitudinal antibody titers into HSAT, SIT, and low titer (LT; |
| Databáze: |
Directory of Open Access Journals |
| Externí odkaz: |
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