Fig. 2.

6MWT and MPS-HAQ results in patients treated with elosulfase alfa after 5 or 6 to 12 months, 1.5 to 2.5 years, and (for 6MWT) 3.5 to 4.5 years. Individual patient responses in 6MWT and MPS-HAQ were classified as no change from baseline, ≥ 10% improvement (longer distance on the 6MWT or lower score on the MPS-HAQ) from baseline, ≥ 10% worsening (shorter distance on the 6MWT or higher score on the HAQ) from baseline, or no measure available. Note that the closest measurement to, but not exceeding, the largest time of treatment was included. For 6MWT, some patients (5, 13, and 31) had normal 6MWT for height [29], and patient 26 had a decrease in 6MWT at 1.5 to 2.5 years because of back surgery (but returned to normal 6MWT for height in later years). 1171A>G: founder pathogenic variant; 6MWT: 6-min walk test; CS: care services; MPS-HAQ: Mucopolysaccharidosis Health Assessment Questionnaire; M: mobility; SC: self-care