Table 1.

Outline of the six ERT scenarios presented in the questionnaire

	Scenario summary	Manifestations		Effect of ERT
		Physical	Intellectual	Physical	Life expectancy	Intellectual
1a	11-year old with severe physical MPSVI and normal intellect, ERT will prolong life	Severe	Nil	Significant improvement	Increased	Nil improvement
2a	MPSI-S with minor health issues as an adult but normal intellect, ERT will only improve health issues a little	Mild	Nil	Minimal improvement	Unchanged	Nil improvement
3a	Severe physical and intellectual manifestations of MPSII. Death in 5 years without ERT	Severe	Severe	Minimal improvement	Increased 3 years	Nil improvement
4a	The 1-year-old younger brother of the MPSII patient in 3a. ERT will prolong his life but not halt the CNS manifestations	Moderate	Severe	Minimal improvement	Increased 15 years	Nil improvement
5a	Mild MPSII with early joint and mobility difficulties, and mild school performance issues	Mild	Mild	Moderate improvement	Not significant	Nil improvement
3b	–a	Severe	Severe	Minimal improvement	Increased 3 years	Significant improvement
4b	–a	Moderate	Severe	Minimal improvement	Increased 15 years	Significant improvement
5b	–a	Mild	Mild	Moderate improvement	Unchanged	Significant improvement

^a3b, 4b, and 5b are identical to 3a, 4a, and 5a; however, the respondents were asked to consider whether they would use ERT for the patients in 3a, 4a, and 5a if it improved the CNS manifestations