Engaging Participants in Rare Disease Research: A Qualitative Study of Duchenne Muscular Dystrophy.

Purpose: Clinical trials in Duchenne muscular dystrophy (DMD) are increasing due to technical advances in genetics, muscle biology and muscle imaging, and translational science. Yet the ability to achieve and measure progress in clinical trials in DMD is severely constrained by recruitment difficulties and low levels of patient and family participation. Clinical trials that do not have full inclusion of patients may affect how well new therapies perform in clinical practice.