Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapy

Characteristic cardiac valve abnormalities and left ventricular hypertrophy are present in untreated patients with mucopolysaccharidosis type VI (MPS VI). Cardiac ultrasound was performed to investigate these findings in subjects during long-term enzyme replacement therapy (ERT) with recombinant hum...

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Veröffentlicht in:	Journal of inherited metabolic disease Jg. 36; H. 2; S. 385 - 394
Hauptverfasser:	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., Wraith, J. E., Barrios, P., Dias da Silva, E., Kurio, G., Richardson, M., Gildengorin, G., Hopwood, J. J., Imperiale, M., Schatz, A., Decker, C., Harmatz, P.
Format:	Journal Article
Sprache:	Englisch
Veröffentlicht:	Dordrecht Springer Netherlands 01.03.2013 Blackwell Publishing Ltd
Schlagworte:	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
ISSN:	0141-8955, 1573-2665, 1573-2665
Online-Zugang:	Volltext
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