Efficacy of avalglucosidase alfa on forced vital capacity percent predicted in treatment-naïve patients with late-onset Pompe disease: A pooled analysis of clinical trials.

Conclusions Models 1 to 3, which had an increased sample size versus COMET, demonstrated a nominally significant effect on FVCpp favoring AVA versus ALG after 1 year of treatment, consistent with results from COMET.

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The relative effect estimated at this time point in COMET was 2.43% (95% CI -0.13; 4.99), which did not reach statistical significance for superiority [ 15 ].

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