Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02).

Overall, patients treated with cipa + mig ( n = 85) showed greater clinically meaningful improvements in absolute and % predicted 6-min walk distance (6MWD) and % predicted forced vital capacity (FVC); however, differences in absolute and % predicted 6MWD did not reach statistical significance, and the difference in % predicted FVC was nominally statistically significant.

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