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Life | Free Full-Text | Pregnancy Outcomes in Late Onset Pompe Disease
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Cells | Free Full-Text | Dysregulation of Metabolism and Proteostasis in Skeletal Muscle of a Presymptomatic Pompe Mouse Model
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In LOPD Patients, 6MWD Test Seen as Reliable Measure of Motor Decline
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Quality of Life with Late-Onset Pompe Disease: Qualitative Interviews and General Public Utility Estimation in the United Kingdom | Published in Journal of Health Economics and Outcomes Research
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Novel approaches to quantify CNS involvement in children with Pompe disease | Neurology
TD-01574-2012_Resolucion-de-fecha-20-12-2012_Art-ii-culo-16-LOPD
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Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe disease | Genetics in Medicine
Frontiers | Immune Tolerance-Adjusted Personalized Immunogenicity Prediction for Pompe Disease
