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
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DOI: 10.1186/s13023-021-01675-x
Abstract: 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… read more here.
Keywords: access agreement; patients mucopolysaccharidosis; treatment; clinical patient ... See more keywords