Mucopolysaccharidosis type VI on enzyme replacement therapy since infancy: Six years follow-up of four children
Clinical and biochemical improvements are reported on Mucopolysaccharidosis type VI (MPS VI) patients on Enzyme Replacement Therapy (ERT) with rhASB (galsulfase, Naglazyme®), and preclinical and clinical studies have shown clinical benefits of early initiation. We report four unrelated MPS VI childr...
Main Authors: | Dafne D.G. Horovitz, Angelina Xavier Acosta, Liane de Rosso Giuliani, Erlane Marques Ribeiro |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2015-12-01
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Series: | Molecular Genetics and Metabolism Reports |
Subjects: | |
Online Access: | http://www.sciencedirect.com/science/article/pii/S2214426915300306 |
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