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Clinical and event-based outcomes of patients with mucopolysaccharidosis VI receiving enzyme replacement therapy in Turkey: a case series

BACKGROUND: The objective of this study was to describe clinical manifestations and events of patients with mucopolysaccharidosis (MPS) VI in Turkey who are treated with galsulfase enzyme replacement therapy (ERT). Clinical data of 14 children with MPS VI who were followed up at the Department of Pe...

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Detalles Bibliográficos
Autores principales:	İnci, Aslı, Okur, İlyas, Tümer, Leyla, Biberoğlu, Gürsel, Öktem, Murat, Ezgü, Fatih
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2021
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8524901/ https://www.ncbi.nlm.nih.gov/pubmed/34666789 http://dx.doi.org/10.1186/s13023-021-02060-4

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