Fig. 3

Pain or discomfort. Respondents were asked to rate the person with alpha-mannosidosis’ pain or discomfort now and 5 years ago on a VAS, where 0 was ‘no pain or discomfort’ and 10 was ‘the worst possible pain or discomfort’. (a) Mean (± SD) change in VAS scores 5 years ago to now for pain or discomfort, stratified by treatment (ERT, HSCT, UP), pediatric (< 16 years old) vs adults (≥ 16 years old) and length of time receiving ERT (< 5 years; ≥5 years); (b) Individual changes in VAS scores for ERT, HSCT, and UP. Green shows improvement, grey no change, and purple worsening/decline; (c) Percentage of patients reporting pain or discomfort now in different parts of the body by treatment: ERT (n = 17), HSCT (n = 6), and UP (n = 10). Respondents could select as many responses as applied. Hence, percentages are calculated as number of patients who chose this option over the number of total patients who responded to this question. ERT = enzyme replacement therapy; HSCT = hematopoietic stem cell transplantation; SD = standard deviation; UP = untreated patients; VAS = visual analogue scale