Effect of alglucosidase alfa dosage on survival and walking ability in patients with classic infantile Pompe disease: a multicentre observational cohort study from the European Pompe Consortium - The Lancet Child &
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Novel GAA Variants and Mosaicism in Pompe Disease Identified by Extended Analyses of Patients with an Incomplete DNA Diagnosis: Molecular Therapy - Methods & Clinical Development
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Correction of oxidative stress enhances enzyme replacement therapy in Pompe disease | EMBO Molecular Medicine
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Cardiac outcome in classic infantile Pompe disease after 13 years of treatment with recombinant human acid alpha-glucosidase - International Journal of Cardiology
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Analysis of LAMPs from control and Pompe iPSC-CMs before and after CQ... | Download Scientific Diagram