Study Summary
RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain. This is a multicenter, open-label dose evaluation clinical study to assess the safety, tolerability, and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in participants with Duchenne.
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RGX-202GENETIC
RGX-202 is a recombinant AAV8 containing a transgene encoding a novel microdystrophin
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | United States |
| Children's Hospital of Orange County | Orange | California | United States |
| Stanford School of Medicine /Division of Neuromuscular Medicine | Palo Alto | California | United States |
| Children's Hospital Colorado | Aurora | Colorado | United States |
| University of Florida | Gainesville | Florida | United States |
| Rare Disease Research | Atlanta | Georgia | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | United States |
| University of Iowa | Iowa City | Iowa | United States |
| University of Kansas Medical Center | Kansas City | Kansas | United States |
| University of Massachusetts Chan Medical School | Worcester | Massachusetts | United States |
| Helen DeVos Children's Hospital | Grand Rapids | Michigan | United States |
| Columbia University Medical Center | New York | New York | United States |
| Cincinnati Children's | Cincinnati | Ohio | United States |
| Nationwide Children's Hospital | Columbus | Ohio | United States |
| Oregon Health & Science University | Portland | Oregon | United States |
| Monroe Carell Children's Hospital at Vanderbilt | Nashville | Tennessee | United States |
| The University of Texas Southwestern Medical Center | Dallas | Texas | United States |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | United States |
| Children's Hospital of Richmond at Virginia Commonwealth University | Richmond | Virginia | United States |
| BC Children's Hospital | Vancouver | British Columbia | Canada |
| Children's Hospital London Health Science Centre | London | Ontario | Canada |
| Children's Hospital of Eastern Ontario | Ottawa | Ontario | Canada |
| The Hospital for Sick Children | Toronto | Ontario | Canada |