The jury is still out. Several companies have high hopes for candidates under development.
Thanks to decades of research, new treatments for muscular dystrophy that address the disease at the generic level may soon change the course of the disease and help more people.
Muscular dystrophy is a rare group of genetic disorders that cause muscles to waste away over time, creating an inability to move and making everyday tasks difficult, depleting quality of life, and causing early death. The disorders are caused by a defective gene located on the X chromosome that controls production of dystrophin, a protein complex found in muscle fibers.