Study Summary
Open-label, single dose clinical trial of scAAV9.U7.ACCA via peripheral limb vein injection for Duchenne muscular dystrophy boys who have a duplication of exon 2.
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scAAV9.U7.ACCABIOLOGICAL
A single dose of scAAV9.U7.ACCA will be systemically delivered via a peripheral vein injection.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Nationwide Children's Hospital | Columbus | Ohio | United States |