My project focuses on late-onset Pompe disease (LOPD), a metabolic neuromuscular disorder characterized by progressive muscle weakness and declining pulmonary function. Since 2006, enzyme replacement therapy (ERT) has been available as a disease-specific treatment and has shown positive effects on several clinical outcomes. However, these benefits often diminish over time and vary widely between patients, for reasons that remain unclear. One hypothesis is that antibody development may play a role in this variability. Additionally, my research explores the potential cardiovascular complications associated with LOPD, which may arise from both direct and indirect disease mechanisms. A further aspect of the project involves evaluating and refining the outcome measures used in Pompe disease, with the goal of improving their accuracy and clinical relevance. My supervisors are prof. dr. Ans van der Ploeg (Erasmus MC, University Medical Center Rotterdam, Center for Lysosomal and Metabolic Diseases, Department of Pediatrics, ) and prof. dr. Pieter van Doorn (Erasmus MC, University Medical Center Rotterdam, Center for Lysosomal and Metabolic Diseases, Department of Neurology).