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Kaplan-Meier survival curve showing comparison of ventilator-free survival CRIM-negative (CN) ERT monotherapy (n = 11) versus CN ERT+ITI (n = 7) treated patients.

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posted on 2013-06-25, 02:27 authored by Suhrad G. Banugaria, Sean N. Prater, Trusha T. Patel, Stephanie M. DeArmey, Christie Milleson, Kathryn B. Sheets, Deeksha S. Bali, Catherine W. Rehder, Julian A. J. Raiman, Raymond A. Wang, Francois Labarthe, Joel Charrow, Paul Harmatz, Pranesh Chakraborty, Amy S. Rosenberg, Priya S. Kishnani

*Three patients in the CN ERT+ITI group began the study invasively ventilated, became ventilator-free with treatment, and are currently still alive and ventilator-free. In contrast, all CN patients in ERT monotherapy treated group were invasive ventilator-free at baseline. This observation suggests that in some cases ERT+ITI can even reverse ventilator dependence in CN Pompe patients.

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