Immune tolerance induction for laronidase treatment in mucopolysaccharidosis I

Immune tolerance induction for laronidase treatment in mucopolysaccharidosis I

Enzyme replacement therapy (ERT) can produce anti-drug antibody (ADA) responses that reduce efficacy or lead to hypersensitivity reactions. Six patients with severe mucopolysaccharidosis type I (MPS I/Hurler syndrome) who did not receive hematopoietic stem cell transplantation underwent an immunosup...

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Bibliographic Details
Main Authors: Roberto Giugliani, Taiane Alves Vieira, Clarissa Gutierrez Carvalho, Maria-Veronica Muñoz-Rojas, Alla N. Semyachkina, Victoria Y. Voinova, Susan Richards, Gerald F. Cox, Yong Xue
Format: Article
Language:English
Published: Elsevier 2017-03-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Mucopolysaccharidosis type I
Enzyme replacement therapy
Immune tolerance
Laronidase
Hurler syndrome
Anti-drug antibodies
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426916301276

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http://www.sciencedirect.com/science/article/pii/S2214426916301276

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