Study Summary
Muscular dystrophies are inherited disorders in which the skeletal and heart muscles become progressively weaker, sometimes leading to permanent disability. Current treatments aim to control symptoms as much as possible, but there is no cure. Gene therapy, in which defective genes causing the disorder are corrected, is a potential treatment option and is in the process of being developed for muscular dystrophies. This study will determine the safety and feasibility of a particular delivery method for gene therapy that could be used in the future to treat people with muscular dystrophies. Only normal saline, and no active treatment, will be used in this study.
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Request More InfoInterventions
Retrograde high pressure transvenous perfusion with normal salineOTHER
Dose escalation of saline volume, infusion rate, and tourniquet pressure, as determined in a stepwise manner and by careful monitoring
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | United States |