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High dose IVIG successfully reduces high rhGAA IgG antibody titers in a CRIM-negative infantile Pompe disease patient

Alglucosidase alfa (rhGAA) has altered the course of an otherwise fatal outcome in classic infantile Pompe disease (IPD), which presents with cardiomyopathy and severe musculoskeletal involvement. However, the response to therapy is determined by several factors including the development of high and...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Veröffentlicht in:Mol Genet Metab
Hauptverfasser: Rairikar, Mugdha, Kazi, Zoheb B., Desai, Ankit, Walters, Crista, Rosenberg, Amy, Kishnani, Priya S.
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2017
Schlagworte:
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5612830/
https://ncbi.nlm.nih.gov/pubmed/28648664
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2017.05.006
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