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Help your MPS VI patients go farther…with NAGLAZYME® (galsulfase)1

Clinical trials of NAGLAZYME have shown promising efficacy and safety/tolerability1,2

A randomized, multicenter, international, placebo-controlled, double-blind, phase 3 clinical trial was conducted enrolling 39 patients* with MPS VI.7

NAGLAZYME Phase 3 clinical Trial Chart

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  • 24-week, double-blind, clinical trial period
  • Patients received weekly treatment with either a 1 mg/kg of body weight
    IV infusion of NAGLAZYME or placebo
  • Patients receiving placebo were switched to NAGLAZYME at the beginning of the open-label extension period. Patients in the drug group received NAGLAZYME for a total of 48 weeks; crossover patients received NAGLAZYME during the 24-week open-label extension period only

The results demonstrated significant clinical benefits, including improved endurance and stair climb.

*One patient in the placebo group left the study for reasons unrelated to treatment.7 Patients receiving placebo were switched to NAGLAZYME in the trial extension period.7

 Next: Improved endurance – walk test

References

  1. NAGLAZYME [Prescribing Information]. Novato, CA: Biomarin; 2005.
  1. Giugliani R, Harmatz P, Wraith JE. Management guidelines for mucopolysaccharidosis VI. Pediatrics. 2007;120:405-418.
  1. Wilcox WR. Lysosomal storage disorders: the need for better pediatric recognition and comprehensive care. J Pediatr. 2004;144(5 Suppl):S3–S14.
  1. Harmatz P, Giugliani R, Schwartz I, et al. Enzyme replacement therapy for mucopolysaccharidosis VI: a Phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study. J Pediatr. 2006;148:533-539.
  1. Wraith JE. The mucopolysaccharidoses: a clinical review and guide to management. Arch Dis Childhood. 1995;72:263–267.