Intravenous Enzyme Replacement Therapy in Mucopolysaccharidoses: Clinical Effectiveness and Limitations

Intravenous Enzyme Replacement Therapy in Mucopolysaccharidoses: Clinical Effectiveness and Limitations

The aim of this review is to summarize the evidence on efficacy, effectiveness and safety of intravenous enzyme replacement therapy (ERT) available for mucopolysaccharidoses (MPSs) I, II, IVA, VI and VII, gained in phase III clinical trials and in observational post-approval studies. Post-marketing...

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Bibliographic Details
Main Authors: Rossella Parini, Federica Deodato
Format: Article
Language:English
Published: MDPI AG 2020-04-01
Series:International Journal of Molecular Sciences
Subjects:
enzyme replacement therapy
ERT
mucopolysaccharidosis
mucopolysaccharidoses
MPS
laronidase
Online Access:https://www.mdpi.com/1422-0067/21/8/2975

Internet

https://www.mdpi.com/1422-0067/21/8/2975

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