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Table 1 NP-C functional disability scale (from [32] and [27])

From: Niemann-Pick disease type C

Ambulation

Score

Language

Score

Normal

1

Normal

1

Autonomous ataxic gait

2

Mild dysarthriad

2

Outdoor assisted ambulation

3

Severe dysarthriae

3

Indoor assisted ambulation

4

Non-verbal communication

4

Wheelchair bound

5

Absence of communication

5

Manipulation

 

Swallowing

 

Normal

1

Normal

1

Slight dysmetria/dystoniaa

2

Occasional dysphagia

2

Mild dysmetria/dystoniab

3

Daily dysphagia

3

Severe dysmetria/dystoniac

4

NG tube or gastric button feeding

4

  1. Abbreviation: NG, nasogastric; a autonomous manipulation; b requires help for tasks but able to feed self; c requires help for all activities; d understandable; e only comprehensible to certain family members.