Neuromuscular Research Center | Phoenix, AZ
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This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to < 18 years
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Interventional model
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22 participants in 1 patient group
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Data sourced from clinicaltrials.gov
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