Impact of long-term elosulfase alfa treatment on clinical and patient-reported outcomes in patients with mucopolysaccharidosis type IVA: results from a Managed Access Agreement in England

BACKGROUND: We present baseline characteristics and follow-up data of a Managed Access Agreement (MAA), including patients with mucopolysaccharidosis IVA (MPS IVA) receiving elosulfase alfa enzyme replacement therapy (ERT) in England on a conditional basis. Patients enrolled in the MAA programme are...

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Foilsithe in:Orphanet J Rare Dis
Main Authors: Cleary, Maureen, Davison, James, Gould, Rachel, Geberhiwot, Tarekegn, Hughes, Derralynn, Mercer, Jean, Morrison, Alexandra, Murphy, Elaine, Santra, Saikat, Jarrett, James, Mukherjee, Swati, Stepien, Karolina M.
Formáid: Artigo
Teanga:Inglês
Foilsithe: BioMed Central 2021
Ábhair:
Research
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC7818902/
https://ncbi.nlm.nih.gov/pubmed/33478511
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-021-01675-x
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