Clinical trial highlights

IGNITE DMD is an ongoing, multicenter, first-in-human open-label Phase I/II ascending dose study of the safety and efficacy of a single intravenous infusion of SGT-001, an AAV9 microdystrophin gene therapy, in DMD patients. DMD is caused by mutations that result in the absence of dystrophin in skeletal and cardiac muscle, which leads to muscle fiber deterioration, progressive decline in motor function, and premature death. SGT-001 microdystrophin is a five-repeat dystrophin surrogate that includes the critical actin, dystroglycan, and nNOS binding domains.

Source: Neuromuscular Disorders - September 19, 2021 Category: Neurology Authors: V. Rao, B. Byrne, P. Shieh, S. Salabarria, J. Berthy, M. Corti, S. Redican, J. Lawrence, K. Brown, C. Shanks, S. Spector, P. Gonzalez, J. Schneider, C. Morris, C. Clary Source Type: research