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NAGLAZYME® (galsulfase) improved endurance: 3-minute stair climb

NAGLAZYME 3-minute climb test

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At 24 weeks:

  • The rate of stair climbing in the group treated with NAGLAZYME was 19.4±4.1 stairs/min at baseline, increasing to 26.8±4.1 stairs/min at week 247
  • The placebo group showed a near constant rate of climb at 30.0±4.8 stairs/min at baseline and 32.6±4.8 at Week 247
  • The difference between the mean change in the rates for the NAGLAZYME and placebo patients was 5.7±3 stairs/min and approached statistical significance in the longitudinal analysis (P=0.053, model-based mean difference)7

At 48 weeks:

  • Patients treated with NAGLAZYME experienced a 38% improvement from baseline at week 24
  • Patients treated with NAGLAZYME experienced a 54% total improvement from baseline at week 487

* All data points shown are group mean difference from baseline. All percentages are calculated as percent change from baseline: group mean improvement as a proportion of group mean baseline value.
† 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; placebo/NAGLAZYME patients received NAGLAZYME during the 24-week open-label extension period only.
‡ One patient in the placebo/NAGLAZYME group failed to complete the assessment.
§For both measures of endurance, the baseline mean established at Week 0 was higher in the placebo group than in the group treated with NAGLAZYME.
One patient in the placebo/NAGLAZYME group left the study before Week 24 for reasons unrelated to treatment.

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References

  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.
  1. Harmatz RP. Mucopolysaccharidosis type VI. Emedicine. December 18, 2006. Available at: http://www.Emedicine.com/PED/topic1373.htm Accessed: November 4, 2008.