The aim of this analysis is to characterize the demographic and clinical characteristics of infantile-onset (IOPD) and late-onset Pompe disease (LOPD) patients who switched from alglucosidase alfa (ALG) to avalglucosidase alfa (AVA) enrolled in the ongoing international, observational, voluntary Pompe Registry (NCT00231400).
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Dimachkie et al. (2024) studied this question.
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