Table 5.

Untreated Arm Functional Outcomes

Endpoints	Baseline	Week 144
6MWT distance	n = 13	n = 6
Mean (SD)	455.1 (51.1)	278.7 (188.9)
Range	351–539	0–525
FVC%p	n = 13	n = 5
Mean (SD)	97.9 (18.3)	77.5 (18.6)
Range	60.85–120.51	53.86–99.96

Genotypes in the untreated arm included (n = 1 each) 1–47, 3–7, 7–17, 8–43, 10–21, 22–25, 30–43, 35–43, 45, 45–50, 46–51, 46–52, 51, and 61–62.