Table 1 Demographics of patients who answered to the questionnaire

Participant sex (M:F)

28:31

Age at onset (n = 41)^a,b,c

42.4 ± 10.3

Age at onset of muscle weakness (n = 53)^a,b

40.4 ± 10.0

Age at onset of Paget's disease (n = 13)^a,b

48.2 ± 10.9

Age at onset of dementia (n = 7)^a,b

56.7 ± 12.5

Age at diagnosis (n = 55)^a,b

46.6 ± 10.5

Age at submission^b

51.7 ± 11.1

Racial categories (n = 55)^a

 Caucasian

52 (94%)

 Asian

2 (4%)

 Black or African American

1 (2%)

Ethnicity (n = 44)^a

 Hispanic or Latino

4 (9%)

 Others

40 (91%)

Mutations in VCP gene (n = 33)^a

 464G>A (R155H)

14 (42%)

 463C>T (R155C)

6 (18%)

 475C>T (R159C)

4 (12%)

 277C>T (R93C)

3 (9%)

 476G>A (R159H)

3 (9%)

 572G>A (R191Q)

2 (6%)

 374G>A (G125D)

1 (3%)