What is muscular dystrophy (MD)?     

Muscular dystrophy is an inherited disease characterized by progressive degeneration of skeletal muscles. Severe muscle degeneration cripples both children and adults and in some forms, takes their lives as a result of the failure of the respiratory muscles. Presently there is no cure.  

What are the different types of muscular dystrophy, and whom do they affect?

There are several types of muscular dystrophies, some of which include Duchenne, Becker, and limb-girdle. Duchenne muscular dystrophy (DMD) is the most severe form; DMD cripples 15,000 North American children and kills two each day. Progressive degeneration destroys the children's ability to walk and to breathe. These children almost never reach adulthood. This disease affects only males since the gene is linked on the X chromosome. Females are only carriers and do not display the symptoms of DMD. In Becker muscular dystrophy (BMD) and Limb Girdle muscular dystrophy (LGMD), the progression of the disease occurs slower than Duchenne.  

Efficacy & Stability

Dystrophin is found in the myoblast-injected DMD muscle  B,D)

at 6 yr after MTT, but not  in the sham-injected control ( A,C ).

E&F are  normal controls.