Duchenne Muscular Dystrophy is the most common fatal genetic disorder to affect children around the world. The first patient, a six year old boy, received, in November 2021, an infusion of DT-DEC01, a novel cell therapy containing Dystrophin Expressing Chimeric Cells (DEC), in Phase I pilot clinical study for the treatment of Duchenne muscular dystrophy (DMD). […]
Progress and limitation of therapeutic approaches targeting DMD By: Dr. Maria Niu Duchenne muscular dystrophy (DMD) is an X—a chromosome-linked recessive disorder caused by mutations in the gene coding muscle cytoskeletal protein dystrophin. Because of the X-linked inheritance, nearly 1 in 3,500 males are affected worldwide, while females occasionally develop symptoms. There is no curative […]