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Novel approach to idursulfase and laronidase desensitization in type 2 and type 1 S mucopolysaccharidosis (MPS)

BACKGROUND: Idursulfase and laronidase are drugs used to treat Hunter syndrome (mucopolysaccharidosis type 2) and Scheie syndrome (mucopolysaccharidosis type 1 S), respectively. These are rare lysosomal storage disorders, leading to accumulation of glycosaminoglycans within lysosomes. Failure of ear...

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Detalles Bibliográficos
Autores principales: Spataro, Federico, Viggiani, Fabio, Macchia, Domenico Giorgio, Rollo, Valentina, Tummolo, Albina, Suppressa, Patrizia, Sabba’, Carlo, Rossi, Maria Pia, Giliberti, Lucia, Satriano, Francesco, Nettis, Eustachio, Di Bona, Danilo, Caiaffa, Maria Filomena, Fischetto, Rita, Macchia, Luigi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9635105/
https://www.ncbi.nlm.nih.gov/pubmed/36329518
http://dx.doi.org/10.1186/s13023-022-02556-7