All Patients (n=140) Received ERT (n=108) Never Received ERT (n=32)
Alive, n (%)a 122 (87.1) 100 (92.6) 22 (68.8)
Last documented age, years (alive)
Mean (SD) 11.5 (7.7) 11.3 (6.4) 12 (12.4)
Median (range) 9.8 (0.6-62.5) 9.9 (0.8-28.8) 8.0 (0.6-62.5)
Deceased, n (%) 18 (12.9) 8 (7.4) 10 (31.3)
Last documented age, years (deceased)
Mean (SD) 12.5 (4.9) 15.1 (3.9) 10.4 (4.8)
Median (range) 13.4 (3.3-20.2) 15.3 (9.1-20.2) 12.2 (3.3-16.2)
Cause of death, n (%)
Related to MPS II 7 (38.9) 3 (37.5) 4 (40.0)
Unclear if related to MPS II 5 (27.8) 2 (25.0) 3 (30.0)
Unknown 6 (33.3) 3 (37.5) 3 (30.0)
Age at diagnosis, yearsb n=136 n=105 n=31
Mean (SD) 3.8 (5.8) 3.5 (3.4) 4.8 (10.4)
Median (range)c,d 2.8 (-0.1-59.7) 2.8 (-0.1-17.8) 2.8 (0.2-59.7)
Cognitive impairment, n (%) 87 (62.1) 69 (63.9) 18 (56.3)
Family history of MPS II, n (%)e 53 (37.9) 40 (37.0) 13 (40.6)
Sibling(s) with MPS IIf 33 (62.3) 22 (55.0) 11 (84.6)
Uncle(s) with MPS IIf 15 (28.3) 12 (30.0) 3 (23.1)
Ethnicity, n (%)
Caucasian 76 (54.3) 54 (50.0) 22 (68.8)
Black/African American 25 (17.9) 18 (16.7) 7 (21.9)
Hispanic/Latino 21 (15.0) 20 (18.5) 1 (3.1)
Mixed 7 (5.0) 6 (5.6) 1 (3.1)
Asian 5 (3.6) 4 (3.7) 1 (3.1)
Other 5 (3.6) 5 (4.6) 0 (0.0)
Unknown 1 (0.7) 1 (0.9) 0 (0.0)
Insurance status
Private 58 (41.4) 43 (39.8) 15 (46.9)
Medicaid 57 (40.7) 46 (42.6) 11 (34.4)
Multi-insurance 13 (9.3) 10 (9.3) 3 (9.4)
Other 9 (6.4) 7 (6.5) 2 (6.3)
Unknown 2 (1.4) 2 (1.9) 0 (0.0)
None 1 (0.7) 0 (0.0) 1 (3.1)