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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Опубликовано в: :JCI Insight
Главные авторы: 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.
Формат: Artigo
Язык:Inglês
Опубликовано: American Society for Clinical Investigation 2016
Предметы:
Clinical Medicine
Online-ссылка: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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