Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome.

@article{Hendriksz2016LongtermEA,
  title={Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome.},
  author={C. Hendriksz and R. Parini and M. Alsayed and J. Raiman and R. Giugliani and Martha L Solano Villarreal and J. Mitchell and B. Burton and N. Guelbert and F. Stewart and D. Hughes and K. Berger and P. Slasor and R. Matousek and E. Jurecki and A. Shaywitz and P. Harmatz},
  journal={Molecular genetics and metabolism},
  year={2016},
  volume={119 1-2},
  pages={
          131-43
        }
}
Long-term efficacy and safety of elosulfase alfa enzyme replacement therapy were evaluated in Morquio A patients over 96weeks (reaching 120weeks in total from pre-treatment baseline) in an open-label, multi-center, phase III extension study. During this extension of a 24-week placebo-controlled phase III study, all patients initially received 2.0mg/kg elosulfase alfa either weekly or every other week, prior to establishment of 2.0mg/kg/week as the recommended dose, at which point all patients… Expand
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