Odiparcil, a potential glycosaminoglycans clearance therapy in mucopolysaccharidosis VI—Evidence from in vitro and in vivo models

Mucopolysaccharidoses are a class of lysosomal storage diseases, characterized by enzymatic deficiency in the degradation of specific glycosaminoglycans (GAG). Pathological accumulation of excess GAG leads to multiple clinical symptoms with systemic character, most severely affecting bones, muscles...

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Detalles Bibliográficos
Autores principales: Entchev, Eugeni, Jantzen, Ingrid, Masson, Philippe, Bocart, Stephanie, Bournique, Bruno, Luccarini, Jean-Michel, Bouchot, Andre, Lacombe, Olivier, Junien, Jean-Louis, Broqua, Pierre, Tallandier, Mireille
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2020
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7228089/
https://www.ncbi.nlm.nih.gov/pubmed/32413051
http://dx.doi.org/10.1371/journal.pone.0233032

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7228089/
https://www.ncbi.nlm.nih.gov/pubmed/32413051
http://dx.doi.org/10.1371/journal.pone.0233032

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