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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...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:Mol Genet Metab
Egile Nagusiak: Rairikar, Mugdha, Kazi, Zoheb B., Desai, Ankit, Walters, Crista, Rosenberg, Amy, Kishnani, Priya S.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: 2017
Gaiak:
Sarrera elektronikoa: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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