Table 1.

Key characteristics in ASPIRO study participants from whom muscle biopsy samples were obtained

Characteristic	Participants, n = 15
Age at dosing (range)	8 months–6 years
Dose level of resamirigene bilparvovec (vg/kg)
1.3 × 1014	6/15
3.5 × 1014	9/15
Ventilation support (mean h/day)
Baseline	22.5
Week 24	12.7
Week 48	6.7
Biopsy samples available (time point)
Left gastrocnemius (baseline)	15/15
Right gastrocnemius (week 24)	15/15
Vastus lateralis (week 48)	14/15
Transgene MTM1 mRNA levels, median (min-max)
Resamirigene bilparvovec 1.3 × 1014 vg/kg
Baseline	0
Week 24	55.6 (10.2–78.9)
Week 48	50.2 (6.5–113. 6)
Resamirigene bilparvovec 3.5 × 1014 vg/kg
Baseline	0
Week 24	128.2 (9.85–322.1)
Week 48	90.5 (132.3–304.6)

Transgene MTM1 mRNA levels are shown as a ratio relative to a reference gene (HEATR6).