Persistence of high sustained antibodies to enzyme replacement therapy despite extensive immunomodulatory therapy in an infant with Pompe disease: Need for agents to target antibody-secreting plasma cells()

With the advent of enzyme replacement therapy (ERT) with alglucosidase alfa (rhGAA, Myozyme®) for Pompe disease, the clinical course of the disease has changed. We have previously described the poor outcome in cross reactive immunologic material (CRIM)-negative and high-titer CRIM-positive (HTCP) pa...

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Main Authors: Banugaria, Suhrad G., Patel, Trusha T., Mackey, Joanne, Das, Stuti, Amalfitano, Andrea, Rosenberg, Amy S., Charrow, Joel, Chen, Y.-T., Kishnani, Priya S.
Formato: Artigo
Idioma:Inglês
Publicado em: 2012
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3711263/
https://ncbi.nlm.nih.gov/pubmed/22365055
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2012.01.019
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