Table 3 Summary of caregiver perspectives on specific hypothetical delays in motor function declines

Agreed that slowing overall physical disease progression is meaningful (N = 12)

10 (83)

Agreed that slowing of progression as defined by no more than a 2-category increase in GMFC-MLD after 2 years is meaningful, if baseline is category 1 or 2 (N = 11)

11 (100)

Agreed that slowing of progression as defined by no more than a 2-category increase in GMFC-MLD after 2 years is meaningful, if baseline GMFC-MLD category was 3 or greater (N = 12)

3 (25)

GMFC-MLD category considered most valuable to preserve for longer before further disease progression (N = 12)

 Unspecified

 1

 2

 3

 4

 5

 6

1 (8)

0 (0)

8 (67)

2 (17)

1 (8)

0 (0)

0 (0)