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

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

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▼ 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 at pharmacovigilance@ptcbio.com.

Registration conditions differ internationally, and prescribing information may vary depending on local approval in each country. The European Summary of Product Characteristics for ataluren is available here. Please refer to your local country guidance for more information.

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