MiscellaneousCardiac Findings After Enzyme Replacement Therapy for Mucopolysaccharidosis Type I
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Identification of a novel fusion Iduronidase with improved activity in the cardiovascular system
2022, Molecular Genetics and Metabolism ReportsCitation Excerpt :In particular, cardiac valvular disease is common in mucopolysaccharidosis type I (MPS I), type II (MPS II), and type VI (MPS VI) [5–12]. While HCT and ERT are therapeutic options for some MPS diseases, HCT and ERT do not treat all cardiovascular complications, specifically cardiac valvular disease [5,13–23]. While the exact reasoning is unclear, the limited benefits of HCT and ERT on cardiac valves is thought to be due to the poor vascularization of cardiac valves [5].
Cardiac manifestations and effects of enzyme replacement therapy for over 10 years in adults with the attenuated form of mucopolysaccharidosis type I
2020, Molecular Genetics and Metabolism ReportsLong-term cognitive and somatic outcomes of enzyme replacement therapy in untransplanted Hurler syndrome
2017, Molecular Genetics and Metabolism ReportsThe factors affecting lipid profile in adult patients with Mucopolysaccharidosis
2017, Molecular Genetics and Metabolism ReportsCitation Excerpt :Haematopoietic stem cell transplantation (HSCT) and enzyme replacement therapy (ERT) have changed the previously life-limiting natural history of the MPSs and improved their survival well into adulthood. Although the positive effect of HSCT and ERT on ventricular function, with no effect on cardiac valve pathology, has been previously recognized [24,25,26,27], little is known about hyperlipidaemia as a potential cardiovascular risk factor in this cohort of patient. Baseline cholesterol data in adults are scarce, reported only for MPS I [28] where it was found to be normal.
Natural history of cardiac findings in mucopolysaccharidosis type I: Report from an international registry
2023, Cardiology in the YoungAirway and Anaesthetic Management of Adult Patients with Mucopolysaccharidoses Undergoing Cardiac Surgery
2024, Journal of Clinical Medicine