Home | Neuromuscular | Duchenne muscular dystrophy (DMD) | Publications | Publication: Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim analysis

Publication: Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim analysis

Published in the Journal of Neurology, this is an updated interim report from the ongoing Strategic Targeting of Registries and International Database of Excellence (STRIDE) international, multicenter registry of real-world ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD)

Review the STRIDE patient characteristics and ataluren safety data
Compare the effectiveness of ataluren plus standard of care (SoC) in STRIDE vs SoC alone in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG DNHS)
Understand the impact of real-world, long-term use of ataluren in patients with nmDMD

▼ 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

Mercurio E, Osorio AN, Muntoni F, et al. J Neurol. 2023;1–18; doi: 10.1007/s00415-023-11687-1

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MED-ALL-DMD-2300094 | June 2025

Publication: Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim analysis

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