Muscular dystrophy (MD) is a group of rare genetic diseases characterized by progressive muscle degeneration and weakness. Characterizing disease progression in the most common form of MD, Duchenne muscular dystrophy (DMD), is a nuanced effort. To evaluate treatment efficacy in DMD clinical trials, QualityMetric adapted typical cognitive debriefing methods to measure disease progression in DMD children using videos and rating scales. In a free case study, learn more about the DMD Video Assessment and its ability to support high-quality, reliable and consistent assessments of patients with DMD.
THIS CASE STUDY WILL DISCUSS:
A novel approach to measuring disease progression in DMD, the DMD Video Assessment requires a parent or caregiver to record six short videos of children performing typical daily physical activities. This case study discusses several aspects of the performance-based assessment, including the relevance of activities measured, the feasibility of the procedures involved and the usability of the accompanying smartphone app.