Duchenne muscular dystrophy, a degenerative disease mostly affecting young boys, is one of the top targets for gene therapy developers. Recently, however, the pace of research has slowed as reports of a worrisome side effect in a number of clinical trial participants have led to study halts and new questions.
Now, scientists may have some strong clues to the cause, due to an unusual collaboration between rival drugmakers competing to develop the first Duchenne gene therapy.
