Table 3.

Characteristics of patients (including patient respondents and children of parent respondents) (n = 181)

Characteristic	n (%) or median (range)
Diagnosed after birth	159 (87.8%)
0–6 months	35 (19.3%)
7–36 months	72 (39.8%)
37–60 months	25 (13.8%)
> 60 months	26 (14.4%)
Unknown age	23 (12.7%)
Current age (years)	10 (0–58)
Race/ethnicity
White	141 (77.9%)
Multiracial	18 (9.9%)
Asian	11 (6.1%)
Other/unknown	6 (3.4%)
Black	2 (1.1%)
Hispanic or Latino	2 (1.1%)
American Indian or Alaska Native	1 (0.6%)
Received postnatal ERT	118 (65.2%)
Received stem cell transplant	49 (27.1%)
Received gene therapy	7 (3.9%)
Clinicaltrials.gov is their main source of information	44 (24.3%)
Type of healthcare coverage
Employer-based	72 (39.8%)
Public (including national healthcare systems)	87 (48.1%)
Privately purchased by a parent	12 (6.6%)
No coverage	4 (2.2%)
Other/Unknown	6 (3.3%)

ERT enzyme replacement therapy