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...

Full description

Saved in:

Bibliographic Details
Published in:	Journal of inherited metabolic disease Vol. 36; no. 2; pp. 385 - 394
Main Authors:	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
Language:	English
Published:	Dordrecht Springer Netherlands 01.03.2013 Blackwell Publishing Ltd
Subjects:	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 Access:	Get full text
Tags:	Add Tag No Tags, Be the first to tag this record!

Be the first to leave a comment!