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
Maureen Cleary(Great Ormond Street Hospital), Karolina M. Stępień(Salford Royal NHS Foundation Trust), Rachel Gould(Birmingham Women’s and Children’s NHS Foundation Trust), Derralynn Hughes(Royal Free London NHS Foundation Trust), Jean Mercer(St Mary's Hospital), James Davison(Great Ormond Street Hospital for Children NHS Foundation Trust), Tarekegn Geberhiwot(Selly Oak Hospital), Saikat Santra(Birmingham Children's Hospital), Elaine Murphy(National Hospital for Neurology and Neurosurgery), Alexandra Morrison, Swati Mukherjee(BioMarin (United Kingdom)), James Jarrett(BioMarin (United Kingdom))
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