Home treatment with Elaprase and Naglazyme is safe in patients with mucopolysaccharidoses types II and VI, respectively

J Inherit Metab Dis. 2008 Dec;31(6):733-7. doi: 10.1007/s10545-008-0980-0. Epub 2008 Oct 19.


Enzyme replacement therapy for lysosomal storage disorders has made an important contribution to improving the quality of life of affected patients. The treatment, however, is invasive and onerous, involving weekly or biweekly intravenous infusions of product over a 3-4 h period. Such therapy can be extremely disruptive of normal family life and the provision of a safe, home treatment regimen is greatly appreciated by affected families. In this report we demonstrate the safety of home treatment with Elaprase for mucopolysaccharidosis type II (17 patients) and Naglazyme for mucopolysaccharidosis type VI (6 patients). Careful patient selection, an experienced home care company and a detailed management plan for potential anaphylaxis and infusion-associated reactions are important components in a successful home treatment programme.

MeSH terms

  • Adolescent
  • Anaphylaxis / prevention & control
  • Child
  • Child, Preschool
  • Home Care Services
  • Home Infusion Therapy / adverse effects*
  • Home Infusion Therapy / methods*
  • Home Nursing
  • Humans
  • Iduronate Sulfatase / administration & dosage*
  • Mucopolysaccharidosis II / drug therapy*
  • Mucopolysaccharidosis VI / drug therapy*
  • N-Acetylgalactosamine-4-Sulfatase / administration & dosage*
  • Recombinant Proteins / administration & dosage
  • Time Factors
  • Treatment Outcome


  • Recombinant Proteins
  • N-Acetylgalactosamine-4-Sulfatase
  • galsulfase
  • Iduronate Sulfatase
  • idursulfase