Age of inclusion: 5-16 Mean age: 9
The drug was well tolerated.
They saw trace dystrophin and reverant fibers pre- treatment.
18 subjects, 9 cohorts… SubQ delivery: .5mg/kg, 1.5mg/kg, 5mg/kg, 8mg/kg, 10mg/kg, 12mg/kg
IV delivery: 1.5mg/kg, 5mg/kg, 12mg/kg
5 weekly injections, 13 week follow up
Increase in dystrophin after 5 weeks: SubQ delivery: 6 of 13 (46%), IV delivery: 6 of 8 (75%)
Higher skipping efficiency than drisapersen
Extension study will use 6-9mg/kg.
No information on timing of next studies were given.