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Table 3 Characteristics of patients (including patient respondents and children of parent respondents) (n = 181)

From: Fetal therapies and trials for lysosomal storage diseases: a survey of attitudes of parents and patients

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%)

  1. ERT enzyme replacement therapy