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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Bibliographic Details
Published in: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.
Format: Artigo
Language:Inglês
Published: American Society for Clinical Investigation 2016
Subjects:
Clinical Medicine
Online Access: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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