Effectiveness of Ataluren in Patients with nmDMD: Confirmatory Evidence from the STRIDE Registry

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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. Methods: Age at LoA was compared between (1) French DYS Registry nmDMD patients and the DYS overall DMD population; (2) CINRG nmDMD patients and CINRG patients with other DMD mutations; and (3) 3:1 propensity-score-matched STRIDE and CINRG nmDMD patients. STRIDE patients were also matched to the combined intention-to-treat populations of three ataluren randomized controlled trials (RCTs), and the 48-week decline in 6-minute walk distance (6MWD) was compared between STRIDE and RCT ataluren-treated or placebo-treated patients. Results: Median ages at LoA were (1) 10.6 years ( n = 43) for DYS nmDMD patients versus 11.1 years ( n = 504) for the DYS overall population; (2) 11.1 years ( n = 16) for CINRG nmDMD patients versus 12.0 years ( n = 382) for CINRG patients with other DMD mutations; and (3) 13.4 years ( n = 48) for STRIDE versus 11.1 years ( n = 16) for CINRG nmDMD patients, indicating a 2.3-year delay in LoA. Mean (SD) 48-week decline in 6MWD was 25.5 (64.8) meters for STRIDE patients versus 25.7 (59.9) meters for RCT ataluren-treated patients and 35.9 (60.2) meters for placebo-treated RCT patients. Conclusion: DMD mutation type did not appear to affect age at LoA. LoA was delayed in STRIDE versus CINRG nmDMD patients, and the 48-week change in 6MWD was consistent between ataluren-treated STRIDE and RCT patients. Mutation type is, therefore, not a source of bias, indicating that STRIDE effectiveness data are reliable. Publication History Article published online: 26 September 2025 © 2025. Thieme. All rights reserved. Georg Thieme Verlag KG Oswald-Hesse-Straße 50, 70469 Stuttgart, Germany

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