Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase.

@article{Harmatz2008LongtermFO,
  title={Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase.},
  author={Paul R. Harmatz and Roberto Giugliani and Ida Vanessa D Schwartz and Nathalie Guffon and Elisa Le{\~a}o Teles and M. Clara S{\'a} Miranda and James Edmond Wraith and Michael Beck and Laila Arash and Maurizio Scarpa and David Ketteridge and John Joseph Hopwood and Barbara R Plecko and Robert W. Steiner and Chester B. Whitley and Paige B. Kaplan and Zi Fan Yu and Stuart J. Swiedler and Celeste Decker},
  journal={Molecular genetics and metabolism},
  year={2008},
  volume={94 4},
  pages={
          469-75
        }
}
UNLABELLED The objective of this study was to evaluate the long-term clinical benefits and safety of recombinant human arylsulfatase B (rhASB) treatment of mucopolysaccharidosis type VI (MPS VI: Maroteaux-Lamy syndrome), a lysosomal storage disease. Fifty-six patients derived from 3 clinical studies were followed in open-label extension studies for a total period of 97-260 Weeks. All patients received weekly infusions of rhASB at 1 mg/kg. Efficacy was evaluated by (1) distance walked in a 12… CONTINUE READING
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