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 two 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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