Treatment outcomes following continuous miglustat therapy in patients with Niemann-Pick disease Type C: a final report of the NPC Registry

Table 1 Modified disability scale

Functional areas	Score
Ambulation
Normal	0
Autonomous ataxic gait	0.25
Outdoor assisted ambulation	0.50
Indoor assisted ambulation	0.75
Wheelchair-bound	1
Manipulation
Normal	0
Slight dysmetria/dystonia	0.33
Mild dysmetria/dystonia	0.67
Severe dysmetria/dystonia	1
Language
Normal	0
Mild dysarthria	0.25
Severe dysarthria	0.50
Non-verbal communication	0.75
Absence of communication	1
Swallowing
Normal	0
Occasional dysphagia	0.33
Daily dysphagia	0.67
Nasogastric tube or gastric button feeding	1

ISSN: 1750-1172