Clinical trial of gene therapy for Duchenne muscular dystrophy underway at University of Florida Health | DuchenneXchange

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Clinical trial of gene therapy for Duchenne muscular dystrophy underway at University of Florida Health


Screening has resumed in a clinical trial to test the effectiveness of a new investigational gene therapy for Duchenne muscular dystrophy. The clinical trial, which began in February with one patient dosed, is being led by University of Florida Health researcher Barry J. Byrne, M.D., Ph.D., who is principal investigator. The Phase I/II trial known as IGNITE DMD is evaluating the safety, tolerability and effectiveness of an investigational gene therapy known as SGT-001 in boys between the ages of 4 and 17.

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