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...

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Publicado en:Mol Genet Metab
Main Authors: Rairikar, Mugdha, Kazi, Zoheb B., Desai, Ankit, Walters, Crista, Rosenberg, Amy, Kishnani, Priya S.
Formato: Artigo
Idioma:Inglês
Publicado: 2017
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Article
Acceso en liña: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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