Observational registry data support ataluren's effectiveness in nmDMD patients, suggesting mutation type may introduce bias.
Background/Purpose: To assess the reliability of ataluren effectiveness data in the Strategic Targeting of Registries and International Database of Excellence (STRIDE [NCT02369731]) nonsense mutation DMD (nmDMD) patients and whether mutation type is a source of bias.STRIDE is an international, observational registry evaluating the long-term effectiveness and safety of ataluren in nmDMD patients. As of 31 January 2023, STRIDE patients had a 3.5-year delay in loss of ambulation (LoA) versus a propensity-score-matched general DMD population from the CINRG Duchenne Natural History Study receiving standard of care alone.
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Werner et al. (2025) studied this question.
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