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An open-label ascending dose cohort study to assess the safety, pharmacokinetics, and preliminary efficacy of neoGAA (GZ402666) in patients with infantile-onset Pompe disease treated with alglucosidase alfa who demonstrate clinical decline or sub-optimal response
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What is the goal of the study?
To learn more about this study, please email the study team member listed below.
Who can participate in the study?
Everyone