Table 2 Participant characteristics^†

Age, mean (SD) [range]

8 (4) [1–15]

40 (6) [26–50]

7 (6) [1–16]

39 (7) [31–50]

 0–2, n (%)

8 (22)

–

3 (25)

–

 3–7, n (%)

7 (19)

–

5 (42)

–

 8–11, n (%)

15 (42)

–

1 (8)

–

 12–17, n (%)

6 (17)

–

3 (25)

–

Gender, n (%)

 Male

23 (64)

7 (19)

5 (42)

1 (8)

 Female

13 (36)

29 (81)

7 (58)

11 (92)

Ethnicity^‡, n (%)

 Arabic

1 (3)

1 (3)

0

0

 Asian

3 (8)

0

1 (8)

0

 Black or African American

4 (11)

3 (8)

2 (17)

1 (8)

  White

24 (67)

28 (78)

11 (92)

11 (92)

  Hispanic

0

0

1 (8)

1 (8)

  Do not wish to answer

5 (14)

5 (14)

0

0

Caregiver relationship to child/adolescent, n (%)

 Parent

–

36 (100)

–

12 (100)

Highest level of education completed, n (%)

 High school, no diploma

–

1 (3)

–

0

 High school diploma

–

7 (19)

–

1 (8)

 Associate's degree

–

5 (14)

–

5 (42)

 Bachelor's degree

–

14 (39)

–

4 (33)

 Graduate degree

–

9 (25)

–

2 (17)

Diagnosis, n (%)

 Before birth

16 (44)

–

6 (50)

–

 At/after birth

20 (56)

–

6 (50)

–

Treatment history, n (%)

 Vosoritide

1 (3)

–

0

–

 Surgery^§

17 (47)

–

8 (67)

–

 None

18 (50)

–

4 (33)

–

Concomitant conditions, n (%)

 Respiratory disease

1 (3)

–

1 (8)

–

 None

35 (97)

–

11 (92)

–