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Table 1 Summary of disease stages

From: Investigating health-related quality of life in rare diseases: a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2)

Disease stage

CLN2 clinical rating scale score (motor score + language score)

Additional characteristics

6

6 (3 + 3)

None

5

5 (2 + 3)

None

4

4 (2 + 2)

None

3

3 (1 + 2)

None

2

2 (1 + 1)

None

1

1 (1 + 0)

None

0

0

None

0 + VL

0

With vision loss

0 + VL + PC

0

With vision loss and requiring palliative care

  1. Disease progression increases with decreasing disease stage
  2. CLN2, neuronal ceroid lipofuscinosis type 2; PC, palliative care; VL, vision loss