Durable and sustained immune tolerance to ERT in Pompe disease with entrenched immune responses

BACKGROUND. Enzyme replacement therapy (ERT) has prolonged survival and improved clinical outcomes in patients with infantile Pompe disease (IPD), a rapidly progressive neuromuscular disorder. Yet marked interindividual variability in response to ERT, primarily attributable to the development of ant...

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Detalhes bibliográficos
Publicado no:JCI Insight
Main Authors: Kazi, Zoheb B., Prater, Sean N., Kobori, Joyce A., Viskochil, David, Bailey, Carrie, Gera, Renuka, Stockton, David W., McIntosh, Paul, Rosenberg, Amy S., Kishnani, Priya S.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Clinical Investigation 2016
Assuntos:
Clinical Medicine
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4970756/
https://ncbi.nlm.nih.gov/pubmed/27493997
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.86821
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