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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Detalles Bibliográficos
Autores principales: Parini, Rossella, Deodato, Federica
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7215308/
https://www.ncbi.nlm.nih.gov/pubmed/32340185
http://dx.doi.org/10.3390/ijms21082975

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7215308/
https://www.ncbi.nlm.nih.gov/pubmed/32340185
http://dx.doi.org/10.3390/ijms21082975

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