| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 10833834 | Molecular Genetics and Metabolism | 2011 | 4 Pages |
Abstract
⺠We report the clinical and immunologic response to ERT in a child with Pompe. ⺠This patient developed anti-rhGAA antibody titers that were blunted yet persistent. ⺠This immunologic response was unlike previously described groups. ⺠The persistence of antibodies likely led to her poor clinical outcome. ⺠Immunomodulatory therapies may be needed in the most severely affected patients.
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Authors
Mary-Alice Abbott, Sean N. Prater, Suhrad G. Banugaria, Susan M. Richards, Sarah P. Young, Amy S. Rosenberg, Priya S. Kishnani,