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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Vydáno v:	Journal of inherited metabolic disease Ročník 36; číslo 2; s. 385 - 394
Hlavní autoři:	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.
Médium:	Journal Article
Jazyk:	angličtina
Vydáno:	Dordrecht Springer Netherlands 01.03.2013 Blackwell Publishing Ltd
Témata:	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
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