Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls
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DOI: 10.1016/j.nmd.2022.02.009
Abstract: Using external controls based on real-world or natural history data (RWD/NHD) for drug evaluations in Duchenne muscular dystrophy (DMD) is appealing given the challenges of enrolling placebo-controlled trials, especially for multi-year trials. Comparisons to external… read more here.
Keywords: placebo; drug; world natural; rwd nhd ... See more keywords