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
Clinical and event-based outcomes of patients with mucopolysaccharidosis VI receiving enzyme replacement therapy in Turkey: a case series
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DOI: 10.1186/s13023-021-02060-4
Abstract: Background The objective of this study was to describe clinical manifestations and events of patients with mucopolysaccharidosis (MPS) VI in Turkey who are treated with galsulfase enzyme replacement therapy (ERT). Clinical data of 14 children… read more here.
Keywords: replacement therapy; turkey; event based; enzyme replacement ... See more keywords