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The importance of integrative disease management

In addition to ERT with NAGLAZYME® (galsulfase), which targets the deficiency of enzyme activity,1 effective MPS VI management may also require palliative treatment administered by a multidisciplinary team of practitioners. Palliative treatment addresses individual complications of MPS VI as they arise, and may include3:

  • Antibiotics and other symptom-based medications
  • Pain management
  • Surgery
  • Adaptive or supportive devices
  • Physical or occupational therapy
Palliative treatment for MPS VI symptoms

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Providing palliative care can also help to proactively address issues that may affect adherence to NAGLAZYME therapy (eg, inability to attend clinic appointments due to illness or hospitalization).

 Next: Improving compliance

REFERENCES

  1. NAGLAZYME [Prescribing Information]. Novato, CA: Biomarin; 2005.
  1. Wilcox WR. Lysosomal storage disorders: the need for better pediatric recognition and comprehensive care. J Pediatr. 2004;144(5 Suppl):S3–S14.
  1. Swiedler SJ, Beck M, Bajbouj M, et al. Threshold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in a survey of subjects with Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). Am J Med Genet A. 2005;134:144–150.
  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. Lachman RS. Radiographic detection of mucopolysaccharidoses (MPS) BioMarin White Paper.
    A guide to understanding Maroteaux-Lamy Syndrome: Mucopolysaccharidosis (MPS) VI. The National MPS Society, Inc.
  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.
  1. Harmatz P, Giugliani R, Schwartz IV, Guffon N, Teles EL, Miranda MC, et al. 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-sulfastase. Mol Genet Metab. 2008. doi:10.1016/j.ymgme.2008.04.001.