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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Article info
Publication history
Published online: January 19, 2023
Accepted:
January 3,
2023
Received in revised form:
December 20,
2022
Received:
October 26,
2022
Identification
Copyright
© 2023 Elsevier B.V. All rights reserved.
