SGCB and Kyphoscoliosis: In preclinical studies, bidridistrogene xeboparvovec gene transfer provided complete restoration of human SGCB protein expression with concomitant improvements in muscle histopathology, kyphoscoliosis, physical activity and cardiac and diaphragmatic function in mice, and no safety issues observed; specifically, diaphragm force production increased by 94% (P < 0.05) and ambulation by 57% (P < 0.05) following treatment compared with SGCB−/− mice20.