104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).

Additionally, nominally significant improvements were reached in the largest pre-specified population (the ERT-experienced group) for both 6MWD and FVC.

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In PROPEL, improvements in the primary endpoint, 6MWD, at week 52 numerically favored cipa + mig versus alglucosidase alfa, but the difference did not reach statistical significance.

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