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Elosulfase alfa in the treatment of mucopolysaccharidosis type IVA: insights from the first managed access agreement

Managed access agreements provide a crucial mechanism whereby real-world data can be collected systematically to reduce uncertainty around available clinical and economic data, whilst providing the opportunity to identify patient sub-populations who are most likely to benefit from a new treatment. T...

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Detalles Bibliográficos
Autores principales:	Stevens, Bob, Kenny, Tom, Thomas, Sophie, Morrison, Alexandra, Jarrett, James, Jain, Mohit
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2021
Materias:	Position Statement
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8467187/ https://www.ncbi.nlm.nih.gov/pubmed/34563214 http://dx.doi.org/10.1186/s13023-021-01876-4

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