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Enzyme replacement therapy interruption in patients with Mucopolysaccharidoses: Recommendations for distinct scenarios in Latin America

BACKGROUND: Mucopolysaccharidoses (MPS) are a group of lysosomal storage disorders, leading to the progressive accumulation of glycosaminoglycans (GAGs) and the subsequent compromising of tissues and organ malfunction. Although incurable, most types of MPS can be treated with enzyme replacement ther...

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Detalles Bibliográficos
Autores principales: Solano, MarthaL., Fainboim, Alejandro, Politei, Juan, Porras-Hurtado, Gloria L., Martins, Ana Maria, Souza, Carolina F. Moura, Koch, Felipe Mendez, Amartino, Hernan, Satizábal, Jose Maria, Horovitz, Dafne D.G., Medeiros, Paula F.V., Honjo, Rachel S., Lourenço, Charles M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7047015/
https://www.ncbi.nlm.nih.gov/pubmed/32140416
http://dx.doi.org/10.1016/j.ymgmr.2020.100572