Table 2.

Untreated patients: Clinical outcomes

	Untreated group
Endpoints	Baseline (n = 20)	Week 96 (n = 9)
6MWT distance, m
Mean±SD	382.6±45.7	252.2±133.1
Range	301.5, 448.0	0.0, 453.5
Ability to rise, n (%)	18 (90.0)	3 (33.3)
Patients with LOA, n (%)	-	1 (5.0)a
NSAA total score
Mean±SD	22.3±7.3	12.0±8.6
Range	12.0, 32.0	1.0, 27.5
FVC%p, %
Mean±SD	96.9±17.7	91.9±14.2
Range	67.5, 125.8	70.5, 113.8

Abbreviations: 6MWT = 6-minute walk test; FVC%p = percent predicted forced vital capacity; LOA = loss of ambulation; NSAA = North Star Ambulatory Assessment; SD = standard deviation. ^an = 20.