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Long-term benefits of physical activity in adult patients with late onset Pompe disease: a retrospective cohort study with 10 years of follow-up.
Home-based enzyme replacement therapy in children and adults with Pompe disease; a prospective study.
Duration of Dual Antiplatelet Therapy for Patients at High Bleeding Risk Undergoing PCI.
Distal muscle weakness is a common and early feature in long-term enzyme-treated classic infantile Pompe patients.
Pathway to diagnosis and burden of illness in mucopolysaccharidosis type VII - a European caregiver survey.