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Case study

Instrument Validation in Rare Disease: Establishing a New Video Assessment as Fit for Purpose in Duchene Muscular Dystrophy

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.

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THIS CASE STUDY WILL DISCUSS:

  • The key progression markers of Duchenne muscular dystrophy
  • The methods of measuring disease progression in children with DMD
  • The validity of using videos and rating scales to monitor physical activity
  • The utility of performance-based video assessments in clinical trials

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.