ABSTRACT
Duchenne muscular dystrophy (DMD) is an important candidate for gene therapy because of its prevalence and life-threatening nature. Although population-based screening programs have been developed for Tay-Sachs disease and cystic fibrosis, this approach is of limited value in DMD since one-third of all cases arise de novo without carrier status in the mother (1,2). Newborn screening is possible and enables early intervention (3-5).
