Clinical outcomes in a subpopulation of adults with Morquio A syndrome: results from a long-term extension study of elosulfase alfa

@article{Hughes2017ClinicalOI,
  title={Clinical outcomes in a subpopulation of adults with Morquio A syndrome: results from a long-term extension study of elosulfase alfa},
  author={D. Hughes and R. Giugliani and N. Guffon and S. Jones and K. Mengel and R. Parini and R. Matousek and S. Hawley and A. Quartel},
  journal={Orphanet Journal of Rare Diseases},
  year={2017},
  volume={12}
}
BackgroundThis post hoc subanalysis examined outcomes in adult patients with Morquio A (mucopolysaccharidosis IVA) who received enzyme replacement therapy (ERT) with elosulfase alfa over a 120-weeks period. Patients ≥18 years of age evaluated in an open-label, long-term extension study of elosulfase alfa (modified per protocol [MPP], n = 32; intent-to-treat [ITT], n = 37; MOR-005; NCT01415427) were compared with the ≥18-year-old untreated population with 2-years follow-up from a Morquio A… Expand
Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance
Mucopolysaccharidosis IVA: Diagnosis, Treatment, and Management
Enzyme replacement therapy for mucopolysaccharidoses; past, present, and future

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