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. 2008 May;152(5):723-7.
doi: 10.1016/j.jpeds.2007.10.015. Epub 2008 Feb 20.

Enzyme replacement therapy for mucopolysaccharidoses: opinions of patients and families

David J Coman  1 , Ian M Hayes, Veronica Collins, Margaret Sahhar, J Ed Wraith, Martin B Delatycki
Affiliations

Enzyme replacement therapy for mucopolysaccharidoses: opinions of patients and families

David J Coman et al. J Pediatr. 2008 May.

Abstract

Objectives: To assess the opinions of individuals with mucopolysaccharidoses (MPS) and their parents regarding the use of enzyme replacement therapy (ERT).

Study design: A validated questionnaire, including hypothetical clinical scenarios about ERT for MPS, was distributed to members of MPS support groups in the United States and Australia.

Results: The questionnaire was completed by 249 MPS support group members. Overall, 92% were in favor of ERT where MPS causes severe physical problems but does not affect intellect, and 69% were in favor of ERT where the physical limitations are mild and intellect is spared. Only 47% were in favor of ERT where severe physical and intellectual problems are well established; however, 77% were in favor of ERT in this situation if treatment begun early prolongs life and improves quality of life.

Conclusion: Most respondents were in favor of ERT for MPS, even where it would not alter the intellectual deterioration. The medical community has a responsibility to advocate for their patients in situations where ERT is appropriate and recognize the economic burden and "family function burden" ERT can incur.

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