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 ClearyJames DavisonRachel GouldTarekegn GeberhiwotDerralynn HughesJean MercerAlexandra MorrisonElaine MurphySaikat SantraJames JarrettSwati MukherjeeKarolina M Stepien

Published in: Orphanet journal of rare diseases (2021)

The MAA data confirm the effects of elosulfase alfa on clinical and PRO results observed in the clinical trials and provide real-world evidence for long-term stabilisation in these measures, suggesting a positive impact on the natural history of MPS IVA.

Keyphrases

patient reported outcomes
replacement therapy
clinical trial
electronic health record
open label
study protocol
phase ii