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Measuring health-related quality of life in Duchenne muscular dystrophy: Current perspectives and recommendations

Published:January 19, 2023DOI:https://doi.org/10.1016/j.jns.2023.120545

      Highlights

      • HRQoL is an important outcome in drug evaluation, clinical practice, and research.
      • A range of instruments has been employed to measures HRQoL in patients with DMD.
      • Yet, in most cases, little is known of their psychometric properties.
      • Recommended measures of HRQoL in DMD include KIDSCREEN, EQ-5D, and HUI.
      • Novel disease-specific tools, such as the DMD-QoL, is pending further validation.

      Keywords

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