Enzymatic replacement therapy for Hunter disease: Up to 9 years experience with 17 patients

Enzymatic replacement therapy for Hunter disease: Up to 9 years experience with 17 patients

Hunter disease is an X-linked lysosomal storage disorder characterized by progressive storage of glycosaminoglycans (GAGs) and multi-organ impairment. The central nervous system (CNS) is involved in at least 50% of cases. Since 2006, the enzymatic replacement therapy (ERT) is available but with no e...

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Bibliographic Details
Main Authors: Rossella Parini, Miriam Rigoldi, Lucia Tedesco, Lucia Boffi, Alessandra Brambilla, Sara Bertoletti, Agata Boncimino, Alessandra Del Longo, Paola De Lorenzo, Renato Gaini, Denise Gallone, Serena Gasperini, Carlo Giussani, Marco Grimaldi, Daniele Grioni, Pamela Meregalli, Grazia Messinesi, Francesca Nichelli, Marco Romagnoli, Pierluigi Russo, Erik Sganzerla, Grazia Valsecchi, Andrea Biondi
Format: Article
Language:English
Published: Elsevier 2015-06-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
MPS II
Hunter disease
Hunter syndrome
Mucopolysaccharidosis type II
ERT
Enzymatic replacement therapy
Idursulfase
Online Access:http://www.sciencedirect.com/science/article/pii/S221442691530001X

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http://www.sciencedirect.com/science/article/pii/S221442691530001X

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