Duchenne Muscular Dystrophy (DMD) is an X-linked genetic disease occurring in 1 in 3,500 births, and is uniformly fatal in boys. It is caused by any one of over 50 known mutations in the gene for dystrophin that result in the production of little to no dystrophin protein a large protein with both structural and signaling capacity that is critical to the normal function of all muscle types. DMD shares many characteristics with other rare diseases that have proven distinctly challenging to drug developers. This presentation will review those issues and illustrate how translational science has, and can, dramatically accelerate the path to potentially life-saving treatments for rare disease patients.