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Long-term safety outcomes and patient preferences for home-based intravenous enzyme replacement therapy (ERT) in Pompe disease and Mucopolysaccharidosis Type I (MPS-I): final results of two-year observation.
Transcatheter Aortic Valve Replacement With Balloon- Versus Self-Expandable Bioprostheses for the Treatment of Bicuspid Aortic Valve Stenosis.
Assessing the health-state utility values of rare disease-hemophilia B using EQ-5D-5L: a study based on the Chinese population.
Langerhans cell histiocytosis of the jaw: clinical analysis of 68 cases.
Caregiver burden and familial impact in Down Syndrome Regression Disorder.