Enzyme replacement therapy in the home setting for mucopolysaccharidosis vi: A survey of patient characteristics and physicians' early findings in the united states

Cynthia Tifft, Virginia Proud, Paul Levy, Kristin DeMarco, Helen Nicely, Sean Turbeville

Research output: Contribution to journalArticle

6 Scopus citations


Galsulfase, a Food and Drug Administrationapproved enzyme replacement therapy for mucopolysaccharidosis VI, is administered once weekly in a hospital setting as a 4-hour intravenous infusion. To improve convenience and alleviate family responsibilities associated with clinic visits, some physicians are transitioning appropriate patients to home infusion therapy. An online survey was conducted with 3 physicians treating 4 patients with mucopolysaccharidosis VI to better understand the factors motivating the transition to home infusion therapy, identify characteristics of appropriate candidates, and evaluate the potential impact on the lives of patients and their families. Survey results showed that home infusion may offer patients and their families increased flexibility of schedule and enhanced family life.

Original languageEnglish (US)
Pages (from-to)45-52
Number of pages8
JournalJournal of Infusion Nursing
Issue number1
Publication statusPublished - Jan 1 2009


ASJC Scopus subject areas

  • Nursing(all)

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