Mucopolysaccharidosis type VI on enzyme replacement therapy since infancy: Six years follow-up of four children

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...

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Bibliographic Details
Main Authors: Dafne D.G. Horovitz, Angelina Xavier Acosta, Liane de Rosso Giuliani, Erlane Marques Ribeiro
Format: Article
Language:English
Published: Elsevier 2015-12-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Mucopolysaccharidosis
Maroteaux–Lamy syndrome
MPS VI
Glycosaminoglycan (GAG)
Enzyme replacement therapy (ERT)
N-acetylgalactosamine-4-sulfatase (ASB)
Galsulfase
Early treatment
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426915300306

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http://www.sciencedirect.com/science/article/pii/S2214426915300306

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