Home | Neuromuscular | Duchenne muscular dystrophy (DMD) | Publications | Publication: Safety and effectiveness of ataluren: Comparison of results from the STRIDE Registry and CINRG DMD Natural History Study

Publication: Safety and effectiveness of ataluren: Comparison of results from the STRIDE Registry and CINRG DMD Natural History Study

This research article, published in the Journal of Comparative Effectiveness Research, outlines the safety and effectiveness of a mutation-specific Duchenne muscular dystrophy (DMD) therapy in 2 propensity score-matched DMD patient populations

Review the safety of a mutation-specific DMD therapy in patients in the Strategic Targeting of Registries and International Database of Excellence (STRIDE) Registry (interim cut-off date: 9 July 2018)
View the effectiveness of this treatment, assessed by comparing outcomes in treated patients with those in a propensity score-matched DMD population from the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), who were comparable in established predictors of disease progression
Age at first symptoms as a measure of disease progression and age at loss of ambulation as a measure of treatment benefit are discussed

▼ Ataluren is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system. Adverse events should also be reported to PTC Therapeutics at pharmacovigilance@ptcbio.com.

Registration conditions differ internationally; always consult local prescribing information and/or Summary of Product Characteristics before prescribing any products.

MED-ALL-ATLN-2500006 | September 2025

Mercuri E, Muntoni F, Nascimento Osorio A et al. J Comp Eff Res. 2020;9(5):341–360

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MED-ALL-DMD-2200190 | June 2025

Publication: Safety and effectiveness of ataluren: Comparison of results from the STRIDE Registry and CINRG DMD Natural History Study

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