Natural history (NH) controls can be attractive in rare diseases such as DMD because they enable smaller drug trials with faster enrollment. Well-known challenges include potential bias due to differences in outcome assessment, and concern that ‘better’ outcomes in clinical trials could arise from more regular follow-up and greater hope for improvement than in NH, especially for effort-based outcomes like 6MWD. To evaluate this potential bias in DMD, we identified clinical trial placebo arms reporting 48-week changes in 6MWD, in meters, and compared these to changes in NH with matched key inclusion/exclusion (I/E) criteria.

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