Study Summary
The purpose of this study is to determine the safety of a miniature dystrophin gene in the treatment of progressive muscle weakness due to Duchenne Muscular Dystrophy (DMD).
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rAAV2.5-CMV-minidystrophin (d3990)BIOLOGICAL
Recombinant adeno-associated virus (AAV) carrying a truncated human dystrophin gene (mini-dystrophin) expressed from a cytomegalovirus (CMV) promoter.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Columbus Children's Hospital | Columbus | Ohio | United States |