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Enrollment status
| Subject ID | Mutation | At gene transfer |
||
|---|---|---|---|---|
| Age (years) | Weight (kg) | Dose | ||
| Subject 1 | duplication exon 22–41 | 8.9 | 37.8 | 2.5 × 1013 vg/kg per leg (5.0 × 1013 vg/kg bilateral total) |
| Subject 2 | nonsense mutation, exon 19 (c.2332C>T/p.Gln778Ter) | 6.9 | 20.9 | 5.0 × 1013 vg/kg per leg (1.0 × 1014 vg/kg bilateral total) |
Enrollment and dosage information for subjects 1 and 2. All AAV vector genome (vg) doses were relative to kilograms of total body weight.
