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Table 2 Comparison of levels of disability, autonomy, and impact on lives between three groups

From: Understanding the challenges, unmet needs, and expectations of mucopolysaccharidoses I, II and VI patients and their caregivers in France: a survey study

Patient characteristics

Group A

Group B

Group C

Caregivers

(nc = 8)

Caregivers (nc = 9)

Patients

(np = 10)

Caregivers (nc = 2)

Patients* (np = 7)

Patient's disability (0 = no disability / 10 = severe disability)

Compared to all patients suffering from the disease

6.7

5.8

6.1

4

2.8 (2)

Compared to all young people of their age

8.8

8.3

7.8

7

5.5 (5)

Patient's autonomy (0 = fully autonomous / 10 = no autonomy at all)

Compared to all patients suffering from the disease

7.7

4.2

4.4

1

1.2 (0)

Compared to all young people of their age

8.5

7.1

6.4

3

1.4 (2)

Impact of disease (0 = no impact / 10 = heavy impact)

Family life

7.6

6.6

6

5

0.6 (0.5)

Professional life

4.7

5.4

7.8

5

1.0 (1.5)

Social life

6.1

4.2

5.9

4

2.8 (3.5)

  1. nc Number of caregivers, np Number of patients
  2. *Values in brackets represent the results from patients who were interviewed in pairs with their caregivers
  3. Group A, Patients not able to answer the survey question because of a severe cognitive impairment (np = 8); Group B, Patients able to answer the survey question with low or no cognitive impairment and high motor disability (np = 10); and Group C, Patients able to answer the survey question with low or no cognitive impairment and low motor disability (np = 7)