Enzyme replacement therapy: efficacy and limitations

Enzyme replacement therapy (ERT) is available for mucopolysaccharidosis (MPS) I, MPS II, MPS VI, and MPS IVA. The efficacy of ERT has been evaluated in clinical trials and in many post-marketing studies with a long-term follow-up for MPS I, MPS II, and MPS VI. While ERT is effective in reducing urin...

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Detalles Bibliográficos
Autores principales: Concolino, Daniela, Deodato, Federica, Parini, Rossella
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2018
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6238252/
https://www.ncbi.nlm.nih.gov/pubmed/30442189
http://dx.doi.org/10.1186/s13052-018-0562-1

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6238252/
https://www.ncbi.nlm.nih.gov/pubmed/30442189
http://dx.doi.org/10.1186/s13052-018-0562-1

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