Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapy - Braunlin, E.; Rosenfeld, H.; Kampmann, C.; Johnson, J.; Beck, M.; Giugliani, R.; Guffon, N.; Ketteridge, D.; Sá Miranda, C. M.; Scarpa, M.; Schwartz, I. V.; Leão Teles, E.; et al. (Journal of inherited metabolic disease, 2013)
Keywords: Adolescent; Adult; Biochemistry; Child; Clinical Trials as Topic; Enzyme Replacement Therapy - adverse effects; Enzyme Replacement Therapy - methods; Female; Heart Valves - drug effects; Human Genetics; Humans; Hypertrophy, Left Ventricular - chemically induced; Internal Medicine; Male; Medicine; Medicine & Public Health; Metabolic Diseases; Mucopolysaccharidosis VI - drug therapy; N-Acetylgalactosamine-4-Sulfatase - adverse effects; N-Acetylgalactosamine-4-Sulfatase - therapeutic use; Original; Original Article; Pediatrics; Randomized Controlled Trials as Topic; Recombinant Proteins - adverse effects; Recombinant Proteins - therapeutic use; Treatment Outcome; Young Adult; Mitral Valve; Enzyme Replacement Therapy; Galsulfase; Aortic Valve; Aortic Regurgitation