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

Overview of attention for article published in Orphanet Journal of Rare Diseases, January 2021

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Open Access UCL Research: 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 https://t.co/Jlm8u9IMPE

18 Feb 2021

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