Algorithm for the Early Diagnosis and Treatment of Patients with Cross Reactive Immunologic Material-Negative Classic Infantile Pompe Disease: A Step towards Improving the Efficacy of ERT

OBJECTIVE: Although enzyme replacement therapy (ERT) is a highly effective therapy, CRIM-negative (CN) infantile Pompe disease (IPD) patients typically mount a strong immune response which abrogates the efficacy of ERT, resulting in clinical decline and death. This study was designed to demonstrate...

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Auteurs principaux: Banugaria, Suhrad G., Prater, Sean N., Patel, Trusha T., DeArmey, Stephanie M., Milleson, Christie, Sheets, Kathryn B., Bali, Deeksha S., Rehder, Catherine W., Raiman, Julian A. J., Wang, Raymond A., Labarthe, Francois, Charrow, Joel, Harmatz, Paul, Chakraborty, Pranesh, Rosenberg, Amy S., Kishnani, Priya S.
Format: Artigo
Langue:Inglês
Publié: Public Library of Science 2013
Sujets:
Research Article
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC3692419/
https://ncbi.nlm.nih.gov/pubmed/23825616
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0067052
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